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UH announces participation in clinical trial testing antibodies to treat COVID-19 in adults – News 5 Cleveland

November 7th, 2020 5:51 am

CLEVELAND University Hospitals announced Thursday its participation in a novel clinical trial testing multiple therapeutics to treat COVID-19 in adults who dont require hospitalization.

University Hospitals said it is adding the ACTIV-2 Trial to its arsenal of investigational treatments. ACTIV-2 is a randomized, blinded, controlled study that tests a variety of new agents against placebo.

This study aims to identify a treatment that can prevent people with COVID-19 from developing advanced disease that requires hospitalization, said Grace McComsey, MD, Vice President of Research and Associate Chief Scientific Officer at UH in a news release. UH is at the forefront of testing experimental treatments for COVID-19, including remdesivir, stem cell therapy and convalescent plasma. Patients suffering from complications of COVID-19 have hope at UH thanks to these options.

This study is one of the few outpatient clinical trials in the Greater Cleveland Area.

There is significant concern nationally as to how hospitals will handle the burden of both influenza and COVID-19 this winter, said Mukesh K. Jain, MD, Chief Academic Officer at UH. Studies focused on helping people recover from COVID-19 at home, without needing hospitalization, are vitally important and we hope our engagement in the ACTIV-2 trial will help us achieve this goal for our community.

The first investigational agent to be evaluated by ACTIV-2 is an experimental monoclonal antibody treatment called LY-CoV555. This antibody was first identified by a blood sample taken from a patient in the U.S. who recovered from COVID-19.

Ones body produces antibodies to fight infections. Monoclonal antibodies are produced in a lab. LY-CoV555 is given as an infusion into a vein in the arm.

To qualify for the study, participants must have tested positive for SARS-CoV-2 infection in the outpatient setting within seven days and started experiencing symptoms within 10 days of enrolling into a study.

For information about enrolling in the trial at University Hospitals:

Call: 1-833-78-TRIALEmail: ClinicalResearch@UHhospitals.orgVisit: uhhospitals.org/researchvolunteer

See complete coverage on our Coronavirus Continuing Coverage page.

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The CDC and the Ohio Department of Health are now recommending the use of cloth face coverings in public to slow the spread of COVID-19.

Read more about the CDC's recommendation here. Here is a step-by-step guide on how to make a face mask from common household materials, without having to know how to sew.

View a global coronavirus tracker with data from Johns Hopkins University.

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UH announces participation in clinical trial testing antibodies to treat COVID-19 in adults - News 5 Cleveland

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Cord Blood Banking Market Technological Innovations And Future Opportunities 202 – Fractovia News

November 7th, 2020 5:51 am

Global cord blood banking market has emerged as one of the most crucial niche verticals of the healthcare industry, driven by the product demand in treatment of many life-threatening diseases including leukemia, thalassemia, and different kinds of cancers. As per The American Academy of Pediatrics, more than 30,000 stem cell transplants had been performed using cord blood by 2013, essentially demonstrating the criticality of stem cell cord blood banking institutions in treating fatal disorders with fewer complications and enhanced medical outcomes.

Request for a sample copy of this report @ https://www.decresearch.com/request-sample/detail/3268

Apart from affirmative policy statements and legislative provisions, the public cord blood banking market is gaining traction due to the fact that some nations such as Italy and France do not permit the establishment of private cord blood banks. Furthermore, an increasing number of funding programs by regional governments to overhaul stem cell cord blood banking research is augmenting the public cord blood banking industry. In fact, as per a research report by Global Market Insights, Inc., the revenue share of public cord blood banking industry is slated to exceed USD 3,931 million by 2025.

One of the main factors supporting the growth potential of public cord blood banking industry is the establishment of legislative provisions that ensure discoveries in the field of regenerative medicine and cellular therapy. In this regard, the Stem Cell Therapeutic and Research Act was established in the U.S. Senate and House Representatives during the year 2015.

India cord blood banking market has registered commendable gains due to the efforts undertaken by various NGOs spread across the country to offer funding for a public cord blood banks, in order to make stem cells available to the Indian population at an economical rate.

Some of the prominent companies in the industry globally include Cordlife, Global Cord Blood Corporation, Singapore Cord Blood Bank, and Jeevan Stem Cell Foundation, among many others.

A considerable increase in the number of patients diagnosed with life-threatening genetic disorders is a major factor supporting enhancements in cord blood banking industry. According to the World Health Organization, approximately 1 in 2000 newborn babies in the European Union are affected by cystic fibrosis. With the increasing prevalence of genetic disorders, the need for cord blood banking is likely to witness enormous growth in the future.

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Partial Chapter of the Table of Content

Chapter 4. Cord Blood Banking Market, By Type of Bank

4.1. Key segment trends

4.2. Public

4.2.1. Market size, by region, 2014 2025 (USD Million)

4.3. Private

4.3.1. Market size, by region, 2014 2025 (USD Million)

Chapter 5. Cord Blood Banking Market, By Services

5.1. Key segment trends

5.2. Processing

5.2.1. Market size, by region, 2014 2025 (USD Million)

5.3. Storage

5.3.1. Market size, by region, 2014 2025 (USD Million)

5.4. Others

5.4.1. Market size, by region, 2014 2025 (USD Million)

Chapter 6. Cord Blood Banking Market, By Application

6.1. Key segment trends

6.2. Cancer

6.2.1. Market size, by region, 2014 2025 (USD Million)

6.3. Blood Disorders

6.3.1. Market size, by region, 2014 2025 (USD Million)

6.4. Immuno-deficiency disorders

6.4.1. Market size, by region, 2014 2025 (USD Million)

6.5. Metabolic disorders

6.5.1. Market size, by region, 2014 2025 (USD Million)

6.6. Bone marrow failure syndrome

Browse full table of contents (TOC) of this report @ https://www.decresearch.com/toc/detail/cord-blood-banking-market

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Cord Blood Banking Market Technological Innovations And Future Opportunities 202 - Fractovia News

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Ashley Cain fights back tears as he reveals 12-week-old daughter will need bone marrow transplant in – The Sun

November 7th, 2020 5:51 am

ASHLEY Cain fought back tears as he revealed that his 12-week-old daughter Azaylia will need a bone marrow transplant regardless of how well her chemotherapy goes.

The 30-year-old star told fans that his baby girl had been diagnosed with Leukaemia last month - just eight weeks after he and girlfriend Safiyya Vorajee welcomed her into the world.

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The devoted dad has been keeping fans updated on Azaylia's battle on Instagram, and couldn't hide his upset as he bravely detailed their latest "crushing blow".

Speaking from his daughter's hospital room while she slept, Ashley said: "I was having a great morning with my little one then the doctors came in and said that no matter how well she does with these blocks of chemotherapy, she's on the high risk list so she'll 100% need a bone marrow transplant.

"And due to her ethnicity, Caribbean, Burmese, Indian, English, it's very difficult to find a match for a transplant.

"Even if she does find a transplant, she's so small and because her case is so bad it's a very, very long, gruelling, and intense procedure and we could be here for a very, very long time - and that's if she manages to fight it and withstand it."

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With his eyes filling with tears, Ashley continued: "It's sad, it feels like we are back to the start - it's broken my heart again.

"It's difficult, it's so difficult. As long as she has a fighting chance we are going to fight and this little warrior, she can do it."

He added: "Please keep us in your thoughts and prayers and please keep sending us positive energy because we are feeding off it."

The star shared some more detail in the video's caption, writing: "Her case is very rare and aggressive and made even rarer by her age.

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"The bone marrow transplant it is a very long and gruelling process of intense chemotherapy, stem cell treatments, transfusions etc and there are many high risk complications.

"I cant lie its a crushing blow as shes in the high risk category and this process is incredibly enduring on her little body. But as always were keeping things positive and as long as shes got a fighting chance WE WILL FIGHT!"

On his Instagram Story, Ashley shared heartbreaking videos of his little girl giggling in her bed, while explaining that she is starting to lose her hair due to gruelling chemotherapy.

The Ex On The Beach star previously called his baby his "little soldier", telling fans: "As long as there's a glimmer of hope, it doesn't matter how much or how little, we will hold on to that and use that to fuel us to the stars and back."

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And sharing the devastating news of his newborn's diagnosis in October, Ashley had written: "Yesterday I had to go through something no parent in the world should have to go through.

"The single most upsetting, terrifying and heartbreaking experience I have ever been through. I found out my beautiful daughter Azaylia Diamond Cain got diagnosed with a very rare and aggressive form of leukaemia, which has come with many complications.

"Everybody who wishes to, please say a prayer for my beautiful brave little girl Azaylia who is currently fighting a battle for her life!

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BEAR NECESSITIESLiam Payne says he was forced to take time apart from Bear during lockdown

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"Mummy and Daddy love you princess LETS GO CHAMP You CAN and you WILL beat this! "

Just one month earlier, Ashley had gushed over how much he loves fatherhood as he thanked his little girl for all that she'd brought him in such a short space of time.

He shares his daughter with long-term girlfriend Safiyya, and the pair tend to keep their relationship out of the limelight.

She previously thanked fans for their support after news of their family's pain broke, as she too vowed that they will all beat the disease together.

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Ashley Cain fights back tears as he reveals 12-week-old daughter will need bone marrow transplant in - The Sun

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‘We’re truly blood brothers’: Stanford coach David Shaw and his recent fight to save his brother, Eric – KGO-TV

November 7th, 2020 5:51 am

David Shaw walks into the hospital room and takes a seat next to the bed. He does this nearly every day, right around lunchtime.

He looks at his younger brother, Eric, tubes snaking across his arms, machines beeping and whirring. Eric does not look like Eric anymore, his skin darkened, scars deepened, features altered. They both know this but never mention it.

Eric is dying, a rare, aggressive skin cancer rampaging through his body with such ferocity that his doctors are nearly out of options. Radiation failed. Chemotherapy failed. Two bone marrow transplants failed.

As Stanford's head football coach, David Shaw is relied on to always know what to say, how to say it and when to say it; but he cannot find the words now that he and his brother are staring down what seems to be an inevitable fate.

"What do you say, where you think you've pulled at the last thread and there are no more threads?" David said. "All I could tell him was that I loved him and that I was there for him. The rest of it was really just ... I thought it was only a matter of time before he passed away."

Two years later, what happened between David and Eric remains real, present and raw -- changing their entire relationship, redefining what it means to be a brother. The words are still difficult to say, so they tip-toe around the crushing physical and mental toll Eric's cancer took on them.

David and Eric are sure to think about it all this weekend, when Stanford opens its season at Oregon on Saturday. Because the last time the Cardinal visited Eugene, neither one knew whether Eric would live or die.

After Stanford came from behind to win that game 38-31 in overtime, David delivered a message at the end of his postgame television interview, looking at the camera and saying, "To my brotherEric: I love you." He tapped the lime green pin on his black Stanford sweatshirt before he left the screen.

When Shaw became head coach at Stanford in 2011, it was the culmination of a family journey. His father was a longtime coach there; David played receiver for the Cardinal and eventually returned as an assistant under Jim Harbaugh. The entire Shaw family -- parents Willie and Gay, along with David, Eric and their sister, Tawnya -- all call the Bay Area home.

To this day, David says the day he was introduced as coach was "one of the better days in all our lives."

Yet something started to happen to Eric that no one could quite figure out. That same year, Eric found strange looking spots on his torso. His wife, Crystal, noticed the first one under his arm. Maybe it was eczema, they thought. Then the spots started to spread. He went to the doctor. They prescribed an ointment, but the spots kept popping up, until they covered his entire body. Eventually, tumors started to grow. It looked as if someone had pushed marbles under his skin. Doctors remained confounded. Eric itched uncontrollably, insatiably. His skin itched so badly, it became difficult to put on clothes, shower, sleep and go to work. He eventually needed sleep medication so he could get uninterrupted rest.

Even then, he itched subconsciously, only realizing what happened when he woke up in the morning to find his arms and sheets covered in blood. Some nights, he tried to sleep on his forearms so his body wouldn't touch the sheets, because his skin grew too sensitive to any touch. At one point, he had more than 30 open wounds on his body.

"It's something that's so pervasive and so destructive that a lot of people have mental problems -- you can't do anything without extreme pain," Eric said. "You bleed a lot through the tumors, through the lesions, through the scratching. A lot of people don't survive, really, because of the mental stress that comes with it."

Doctors had a hard time diagnosing his disease because it is often confused with psoriasis, eczema or other skin conditions. Eventually, they determined he had a rare form of skin cancer called mycosis fungoides, a type of T-cell lymphoma that affects one in 6 million people in the United States and Europe. At the time, Eric Shaw was 38.

In 2013, he and Crystal pushed for a referral to Stanford Cancer Center, which has leading experts in the disease. Mycosis fungoides is so rare, it accounts for only 4% of all non-Hodgkin lymphoma cases; among those who suffer from it, only 20% have the type of itching Eric experienced. Rarer still is to find it in people under the age of 40, and African American men often end up with the worst prognosis. All the odds were firmly against him.

"When you first hear skin cancer, your mind doesn't go too far," David said. "So initially I was like, 'There are creams and other minor surgeries. I think it'll be OK.' And then Eric said, 'No, this is not the typical skin cancer. This is inside my body. This is inside the layers of my skin, and it's not one spot. It's everywhere.'

"I didn't really get it for weeks after that because, rectifying something that I didn't think was so serious to [then thinking] ... 'Oh my gosh. So this is really cancer. This is really scary now.' It took a long time for that to sink in."

David turned it over in his mind. He was the big brother, the protector, the one who always made sure Eric would be OK. They were supposed to raise their kids together, grow old together, and reminisce about the randomness of a life spent together.

He kept coming back to one thought: You're not supposed to lose your little brother.

David and Eric Shaw grew particularly close as children as they moved from place to place when their father, Willie, took new coaching jobs. Tawnya, their older sister, fit in anywhere socially. But David and Eric, who is two years younger, stuck together.

"Like a pair," David said.

They loved riding their bikes and, when they moved to Arizona, they took advantage of the wide-open spaces in the new development where they lived. They rode for miles and miles, setting up their own ramps and doing tricks and wheelies, visiting friends along the way before returning home after dark. They played sports, too, and though David loved football as much as their dad, the basketball court is where the brothers had their epic battles.

"I was always kind of a little bit stronger and I'll never forget the last time we played one-on-one basketball," David said. "He just got better than me, and he won, and once I got over the anger and disappointment, I was proud because my younger brother had grown and was gaining confidence."

Said Eric: "I wanted nothing more than to beat him, and he wanted nothing more than to keep beating me. But, during those times, it was just us, it was me and him. He was my best friend."

David went on to play at Stanford and eventually got into coaching, against his mother's best wishes. Eric did not pursue a career in athletics. He went to San Diego State and got into a career in marketing at a financial services company, where his gregarious nature, big smile and easy laugh made him a perfect fit. Though their personalities are different -- David is stoic and introspective, Eric makes anyone feel as if they have been friends forever -- they are grounded in the same values they learned at a young age: family and faith above everything else.

Those principles only grew stronger after they found themselves in the Bay Area as adults.

After David was hired by Stanford, the entire Shaw family made it clear it would always be around to support him. Family members all have a standing invitation to come for dinner on Tuesdays. And they always attend home football games, waving and hugging David during the team's pregame walk, cheering from the stands, and then waiting for some time together once the game ends.

Even as Eric grew sick, he made it a point to go cheer for his big brother. "It's not just the football game. Our family comes together," he said. "We celebrate, we come to watch the game and cheer the team on and support David. And then afterwards, win or lose, we all wait for him to come out. It's a family day. It's been wonderful to share that experience with David."

Stanford eventually drew them even closer, and it had nothing to do with football.

Eric did not understand the gravity of his situation until his first meeting in 2013 with the doctors at Stanford Cancer Center. They put it bluntly: He had such an aggressive form of the disease that he needed immediate treatment. They would start with total skin radiation, preparing Eric to lose his hair, eyebrows, eyelashes, fingernails and toenails.

If that did not work, they would try chemotherapy next.

"All these thoughts are running through your mind," Crystal said. "'Is he going to make it? Is it going to work? What's going to happen?' At the time, our youngest daughter was 3 months old, so it was pretty overwhelming. We were just putting our lives together and then boom: you're in the middle of this cancer war."

The next week, Eric took a leave of absence from work and began four-times-a-week trips from their home east of Palo Alto, California, to Stanford Hospital, often driving as many as three hours one way in traffic. When he arrived, he went into a box and his whole body was exposed to the radiation light for about an hour. Then, he would make the drive back home to see Crystal and their four kids -- Caleb Michael, Jared Spann-Shaw, Madison Shaw and Olivia Shaw.

The radiation charred his skin. He lost weight. When he looked in the mirror, Eric no longer recognized the man looking back at him.

"Nothing prepares you for something like this," he said. "Knowing that other people were looking at me and knowing that something was very wrong, that was a daily grind to get myself up out of bed and get ready for the day, knowing that that was going to be my life."

He did this for three straight months, all to keep the disease from growing to a point where it would kill him. It worked for a short time, but the disease came back more aggressively six months later. Doctors moved on to chemotherapy treatments, some of them experimental, but also began discussing the last-resort option: a bone marrow transplant.

David and Tawnya immediately volunteered to become donors, and underwent testing. In most cases, siblings are the best chance at a donor match. Unfortunately, in their case, neither was close. On a 10-point match scale, Tawnya registered a 3, David a 5. Neither qualified to donate.

"I wanted to jump to the front of the line and say, 'Whatever I have to do, whatever you have to take out of me, however you have to do it, just do it,'" David said. "For them to come back and say that you're not a strong enough match was disheartening. It hurt me. The fact that we had to put our trust and faith in people that we didn't know, and that we're going to have to go out to registries and try to find someone who was a better match than I was, that uncertainty, and that doubt, it's hard to keep it at bay at that point. It starts to creep in."

Doctors eventually found two donors whom they believed could work, but they were not perfect matches. In early 2018, Eric and his family moved into a two-bedroom apartment near Stanford Hospital to prepare for the transplant. For three months, he went through radiation, then chemotherapy to prepare his body to accept the donor cells.

He underwent the transplant in April, feeling confident and inspired it would work. After a month, doctors did an initial check to see how many of the donor cells had survived the transplant.

None survived.

"It was like I never even had the transplant," Eric said. "That was so devastating. We just knew it was going to work. I mean, we're people of faith, and we knew everybody was praying for us, and that we were praying that this six-year journey was going to finally be over. And it wasn't over. It was crushing for them to say, 'It didn't work. We're going to have to try again.'"

The second attempt happened in September. Crystal bought lime green pins for the family to wear for lymphoma awareness. Without telling Eric or Crystal, David decided he would wear his on his shirt for the 2018 football season. In addition to that, he had lime green and yellow ribbons placed on the back of Stanford helmets as a way to show support for both cancer patients and cancer survivors.

He told his team that his brother was fighting cancer, and briefly mentioned the helmet ribbons publicly during an early-season news conference. But beyond that, David kept the severity of what was happening to his brother to himself, masking his growing nervousness, fear and anxiety as the clock ticked toward the next transplant. He had a hard time processing what was happening. He did not want to put that at the feet of his players, or his staff.

The doctors used the same donor cells that failed the first time for the second transplant on Sept. 11, 2018, because that was the only option available. But this time, doctors used even stronger drugs to prepare Eric's body to receive the donor cells -- hoping that would do enough to stop his immune system from attacking them.

When Stanford played Oregon on Sept. 22, no one in the Shaw family knew whether the transplant had worked. But the situation was more dire than the first transplant. The stronger chemotherapy caused major complications, and Eric became severely ill.

David coached the game with this in the back of his mind. Stanford rallied from a 21-7 deficit to win an overtime thriller, moving to 4-0 on the season, with a top-10 matchup against Notre Dame the following week. Back in Palo Alto, Eric watched the entire game alone in an apartment he rented near the hospital, the comeback buoying his spirits.

He had no idea his brother would speak to him through the television until he heard the words, "To my brother Eric ..."

"In that moment, I didn't feel any sickness at all," Eric said. "I can't really describe what I felt, just how proud I am of him and how awesome it made me feel that he would do that for me."

Said David: "If that transplant didn't work, I didn't know how many more games he was going to be able to see. That was an opportunity for me on national TV to speak to him, to say to my brother that against the odds, we came back and throughout the entire game, I was thinking about him."

Eric soon returned to Stanford Hospital. The chemotherapy destroyed his blood system, so he needed daily blood transfusions to stay alive. It came as no surprise when doctors told him the second transplant had failed. They had no plan now, no other donor options. David came by to visit as often as he could, but he had a hard time finding the words to say to his dying brother.

"I thought about Crystal. I thought about their kids," Shaw said. "I thought about, 'How can we help?' And then I kept going, 'We just can't get there. There has to be something else.' And we all prayed and we all comforted each other and trusted the doctors and prayed for the doctors. And just kept saying, 'Just tell us whatever options there are. Just tell us what to do and we'll do it.'"

During the day, Eric had his mother, Crystal, David, or David's wife, Kori, at his side, helping to keep his mind off what was happening to him. But in the evenings, when he was alone in his hospital room, he couldn't help but think about the dwindling medical options and his own death, slowly accepting what he believed would inevitably come.

Over seven years, everything the doctors tried had failed, and the disease always came back more aggressively. He felt exhausted in every possible way, desperate to feel better. He didn't want to die. All he wanted to do was get better, and see his kids again, hug his wife and go home. But that possibility seemed as far off as the stars.

"The doctors couldn't help us," Eric said. "They had lost all hope. There was nothing left, but we were in the deepest part of the valley, and there was nobody there but God. I said, 'You're going to take me off this Earth.' And he told me, 'Eric, you're not going to die.' That was the point at which my faith really took over, and I really had true peace."

His team of doctors huddled together again and came up with a plan many of their colleagues questioned, simply because they had never attempted it. In mid-October of 2018, they told Eric they wanted to try a third transplant.

Only this time, they wanted David to be the donor and they had only weeks to make it happen.

Eric thought, "Are they trying to kill me?"

When David was initially rejected, doctors had worked for 25 years to find a way to do half-match transplants but had virtually no success. By 2018, doctors explained that a different way to do the transplant had emerged, opening up the potential to try it with Eric. These transplants, called haploidentical transplants, typically use donor cells from a family member.

Dr. Wen-Kai Weng, Eric's bone marrow transplant physician, explained, "It was relatively new at this time. We decided to go ahead, because we knew if we didn't do it, the disease would really come back with a vengeance."

No one had ever done a third transplant with donor cells at Stanford.

"If he didn't go for this risk, he wouldn't be here," said Dr. Youn Kim, who treated Eric and heads Stanford's multidisciplinary Cutaneous Lymphoma Clinic/Program. "He wouldn't be living."

Doctors told Shaw there was a 15% chance he would not survive the transplant itself. If he did survive it, there was only about a 30% to 40% chance the donor cells would work. Compared to much steeper survival odds with no transplant at all, the decision -- filled with multiple layers of danger -- did not feel risky at all.

They had to try.

"They might have told us what the odds were, and I honestly just pushed it out of my brain," David said. "If this is the Hail Mary, hey, we're going to drop back and throw it as far as we can and send prayers along with it and hope that it works."

Without hesitation, David said to his brother, "Tell me what I need to do."

Stanford gathered in its team hotel early on Oct. 27 to begin final preparations before hosting Washington State later that day. David checked in for a 9 a.m. meeting and when it finished, he checked out of the hotel without saying a word. He walked toward the back exit, careful to make sure no one saw him, and snuck out the door to a waiting car.

Shaw sat in the passenger seat, headed toward campus and Stanford Hospital, praying all the while that what he was about to do would work.

He arrived at the hospital and was hooked up to an IV for the first dose of medication. This would not be the more traditional bone marrow transplant, where cells are extracted with a needle through the hips. Rather, the medication flowing through the IV would stimulate his body to overproduce the stem cells needed for the transplant, flooding his blood with them. The cells would then be extracted from his blood, and transplanted into Eric.

Doctors told him to expect to start feeling joint pain and tiredness within 24 hours. Those symptoms would grow only stronger over the coming days, when he came in for more medication. They told him he should stay off his feet, rest and remain hydrated.

That would be nice, David thought. But he had a game to coach. Only two people inside the program knew he had gone that morning: assistant athletic director for football operations Callie Dale, who drove him to the hospital, and defensive coordinator Lance Anderson.

"The way that I do my job, I work really hard not to make it about me," David said. "Although I wanted my team to know what my family was going through, college football is about the student-athletes. I wanted them to focus on what they needed to do. I didn't want to pull from that. I didn't want to, all of a sudden, now make it about me and my family."

A few hours later, he returned to the team hotel and acted as if he had been there the entire day, speaking nothing about his trip to the hospital. Shaw put on his lime green pin and made his way toward the bus. The short ride to the stadium felt long that day. His mind wandered before returning to the flip card in front of him.

As he exited the bus and finished the walk to the stadium, his two young nieces ran up to him. They squeezed him, holding on longer than usual, as if they knew their Uncle David was their only option, too.

He worried players would notice him moving around so slowly. If they did, no one said a word. Shaw kept pushing the pain aside, shoving his emotions down deep, saying prayers every chance he got.

On Wednesday, Shaw woke up and was so lethargic, he felt as if he was moving like a sloth. He went to the hospital for the final procedure: extracting the cells from his blood. Shaw wore comfortable clothes, arranged his pillows and settled in for a long day ahead. Doctors hooked him up to a machine that would do the work through two IVs: One took his blood so the needed donor cells could be siphoned out; the other IV would put the blood back in his body.

Eric rested on another floor in the same hospital.

David worked on his game plan, watched a few movies and occasionally stared at his own blood in the IVs, willing it to save his brother. He kept saying to himself over and over again, "God, I hope this works."

After eight hours, he was finished. Shaw then went out to practice.

"I remember walking up to him and just asking him, 'How are you doing, how are you feeling?'" Anderson said. "I could see it in him that he wasn't his normal self. He paused for a little bit and then he's like, 'I'm OK. A little bit tired, but I'm OK.' You know, just trying to put the most positive light that he could on it."

The next day, Nov. 1, 2018, Shaw went back to the hospital. It was transplant day, and he had to be with Eric to witness what they hoped would be a miracle. David and Crystal watched as Eric received a transfusion of David's stem cells, a shimmering light pink fluid flowing into his body. They sang and prayed. Already, they had received one small bit of good news: Doctors extracted 28 million cells from David's blood, about 20 million more than what they had hoped to get.

Stanford traveled the following day to Seattle, for a game against Washington. David felt guilty for leaving, but he knew there was nothing else he could do. Eric struggled in the hospital, not only from the transplant, but from the heavy chemo and radiation doctors used to prepare his body for the new cells.

Eric ran a fever of 105 degrees and vomited for days. The pain grew so intense he was put on a morphine drip and was in and out of consciousness. In Seattle, Shaw remembers being locked into the game, "except for those little moments where my heart was with my brother."

Stanford lost another heartbreaker, 27-23.

"I know us losing had nothing to do with everything David was going through," Dale said. "But just piling that on with everything else he was dealing with, it was a lot for him. He brought that up many times, about how Eric would tell him the biggest excitement for him every week was watching us play and watching us win. I know David had a lot of pressure on himself, amongst the pressure he already has as a head coach, to win for Eric. And I know that every time he did, he really felt like it was for him. And when we came up short, I know he was probably even harder on himself than he normally would have been."

Back at Stanford, David visited Eric when he could. But the waiting game took an increasing mental toll. David prides himself on his ability to compartmentalize, to focus on the only thing in front of him. He never spaces out, and he rarely gets emotional. But Shaw was falling apart on the inside.

He often found himself staring at cut-ups of red zone plays, not realizing the film had been paused for 20 minutes while his mind drifted off. Whenever that happened, he would stop and call someone, either his brother, his wife, his mother or Crystal just to see how they were doing.

"There were times where I thought life was slow motion, but it was actually moving and I was the one who was in slow motion," David said. "I found myself sometimes saying, 'Is this real? Is this really happening? This shouldn't happen.'"

In the middle of every single meeting, in the middle of every single film session, he silently prayed, "God help my brother. Just please let this one work."

"I look back now and I know more of everything that was going on and the situation," Anderson said. "I realized how much he was dealing with and how much he had to bear that week. And it's amazing that he was able to go through that week without really letting any of us really know exactly what he was going through and what a big deal this really was."

Within a few weeks, Eric started to turn a corner. Though they did not know whether the transplant had worked just yet, he showed enough improvement to leave the hospital after 52 days. David arrived for the big day, and Eric slowly put on a protective mask before shuffling to a waiting wheelchair. Doctors, nurses and support staff lined the hallway, clapping and cheering.

David cries when recalling that moment, his pent-up emotions flooding out as he describes it publicly for the first time.

"This is my little brother, after years of cancer, getting to leave the hospital," Shaw said, his voice quavering. He pauses to wipe tears from his eyes. "The nurses were crying. The doctors were crying. Because a few months earlier, they were preparing us for him to die. And he got to go home."

Three days later, doctors met with Eric and Crystal to deliver the results from the transplant. After only 27 days, Eric had none of his own blood coursing through his body.

It was all David's.

Eric picked up the phone.

"Dave," Eric said. "You have a twin. We're truly blood brothers."

Eric, who turns 46 on Friday, has lived a fairly normal life since he was declared cancer free on Jan. 1, 2019, although the coronavirus pandemic has limited how often the Shaw family can see each other.

In September, they decided to get together to celebrate all of their recent birthdays at David's house. They stayed outdoors, socially distanced, with masks on. Eric and David allowed themselves a hug, their heads turned to the side.

"Every time I see him, I just smile, you know? Because he gets to be here," David said.

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'We're truly blood brothers': Stanford coach David Shaw and his recent fight to save his brother, Eric - KGO-TV

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CA Prop 14 Explained: What To Know Before You Vote Election Day – Los Angeles, CA Patch

November 7th, 2020 5:51 am

LOS ANGELES, CA Proposition 14, the only statewide bond measure on the general election ballot, asks voters to authorize $5.5 billion in bonds to fund stem cell research. If that sounds familiar, that's because it is. California approved a similar bond request 16 years ago, allowing the state to prop up what was then a fledgling and controversial area of research.

Prop 14's supporters contend the money is needed to fund cutting-edge research on the brink of discovering treatments and cures that could help save countless lives. Opponents say such promises are "shameless exaggerations" and that California isn't in a position to spend billions on stem cell research.

In 2004, Californians authorized $3 billion in bonds to create the California Institute for Regenerative Medicine, with the aim of making the Golden State a hub of cutting-edge stem cell research. It offset the George W. Bush administration's decision to halt federal funding for embryonic stem cell research. Sixteen years later, the California Institute for Regenerative Medicine is running out of money, forcing it to suspend new projects. Last year, the institute stopped accepting new applications, according to Ballotpedia.

If Prop 14 passes, it will authorize $5.5 billion in state general obligation bonds to support private, university and nonprofit stem cell research and therapy for diseases and conditions such as cancer, HIV/AIDS, Alzheimer's, Parkinson's, strokes, epilepsy and other neurological conditions. In addition to funding research, the measure would help fund treatment and physician training.

The measure caps the California Institute for Regenerative Medicine operating costs at 7.5 percent of the funding, with the rest going to grants. Over the last decade, the bulk of the institute's grants went to California universities and hospitals. It will cost the state about $260 million a year for 30 years to repay the bonds.

According to the text of the measure, the institute has generated more than $3 billion in matching funds, sponsored more than 1,000 research projects and treated thousands of patients. It claims to have promising treatments in the pipeline awaiting funding for final stages of research.

Check Out The CalMatters 2020 Election Guide

"This medical revolution holds the promise of restoring health and quality of life for many of California's individuals and families suffering from chronic disease and injury," Robert Klein, chairman of Americans for Cures, told the California Stem Cell Report blog. "However, the last tactical mile to bring this broad spectrum of therapies to patients will require more funding and the thoughtful support of California's public as the human trials and discoveries are refined and tested, overcome numerous obstacles or complications, and ultimately serve to improve the life and reduce the suffering of every one of us."

Opponents of the measure say that the California Institute for Regenerative Medicine is no longer necessary because the federal government now spends billions to support stem cell research and private entities are leading the way on advancements without the help of taxpayer-funded grants. They question the institution's track record, oversight and budget.

"We can't afford to waste billions. In the middle of an economic crisis, with soaring unemployment and budget shortfalls in the tens of billions of dollars, we don't have money to burn," reads the opposition on the Official Voter Information Guide. "Paying back Prop. 14's costs of $7.8 billion could mean huge tax increases at a time when our economy is on its knees. Or laying off thousands of nurses and other heroes who do the real work of keeping California healthy."

The measure has the support of the California Democratic Party and the University of California Board of Regents. More than $9 million has been spent on the Yes On 14 campaign, while there are no official opposition campaigns. However, several newspaper editorial boards have come out against the measure including The Orange County Register, the Mercury News and The Bakersfield Californian.

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The Many Model Systems of COVID-19 – The Scientist

November 7th, 2020 5:51 am

Earlier this year, as transmission of SARS-CoV-2, the virus behind the COVID-19 pandemic, started to pick up speed, researchers around the world hurried to find model systems that could provide insight into disease spread, host immune responses, and possible treatments.

When the pandemic first started, nobody really knew what was going to be the best model, says Amanda Martinot, a veterinary pathologist at Tufts Cummings School of Veterinary Medicine.

The most widely available candidates were mice, which are easily housed and so well-researched that there are tons of tools available for studying nearly every aspect of their biology. But as researchers suspected, based on previous incompatibility of mice and other coronaviruses, the animals present challenges when it comes to studying SARS-CoV-2. The virus uses a human receptor called ACE2 to get into cells, but mouse ACE2 is different enough that the virus doesnt readily bind it.

Scientists have overcome the issue using two separate strategies: generating transgenic animals that express the human receptor and modifying the SARS-CoV-2 virus to make it better able to bind mouse ACE2.

Even with the success of those approaches, investigators have also been on the lookout for other options. Ferrets, for instance, are useful for studying viral transmission and viral replication in the upper respiratory tract and have been used by several groups for SARS-CoV-2 experiments. Some researchers have also turned to hamsters, which have been used in the past to study other viruses, including the coronaviruses responsible for severe acute respiratory syndrome and Middle East respiratory syndrome.

Hamsters are known in virology for their permissiveness to all kinds of viral infection really, and, as a rule of thumb, I would say if something doesnt work in a mouse, you may as well try a hamster, says Jakob Trimpert, a postdoc at the Free University of Berlin. And if youre lucky, it works there. Along with collaborators, hes been using several species of hamsters to study SARS-CoV-2 infections and potential therapeutics.

The hamster ACE2 receptor is compatible with SARS-CoV-2, and the animals develop clear clinical indicators of disease, he explains. For instance, Syrian hamsters(Mesocricetus auratus), one of the hamster species most commonly used in virus research worldwide, get mild SARS-CoV-2 infections, but their main symptomweight lossis reproducible and possible to measure. These animals also have severe pneumonia that is detectable via lung pathology. Trimpert and his colleagues used both transcriptomics and proteomics to evaluate the animals immune responses to SARS-CoV-2 in a study they published on July 20.

Many alveolar air spaces (white) are collapsed during a SARS-CoV-2 lung infection in a Syrian hamster (right). They are not collapsed in an uninfected hamsters lung (left).

Amanda martinot, tufts cummings school of veterinary medicine

Hamsters have been the best model so far for showing us any clinical disease, says Martinot. They develop weight loss, and they develop a fulminant pneumonia where its affecting sometimes over fifty percent of their lung, she adds. They also will recover if given time, but the pathology we feel is more representative of what you might see in humans. Martinot, Dan Barouch, who directs the Center for Virology and Vaccine Research at Beth Israel Deaconess Medical Center, and other collaborators recently published a study showing that a SARS-CoV-2 vaccine protected against weight loss and pneumonia in Syrian hamsters.

This species, also known as the golden Syrian hamster, has some drawbacks, too, Trimpert says. Theyre big, for one, weighing in at 150160 grams fully grown, in contrast to adult mice, which tip the scales at about 3035 grams. Syrian hamsters are also aggressive toward each other and sometimes the people taking care of them. Their size and aggression mean that they often have to be housed alone in larger cages, which makes them more expensive to keep.

The biggest issues, according to Trimpert, are the poor quality of the sequenced genomemany genes are just missingand the lack of molecular tools that work in hamsters. To circumvent these problems, Trimpert and his colleagues are resequencing and annotating the Syrian hamster genome.

Because the weight loss in Syrian hamsterstheir only easily observable clinical symptomis quite mild, its also a drawback, particularly when testing therapies or vaccines, Trimpert tells The Scientist. If you need thirty hamsters in order to get statistical significance . . . that is a huge practical problem.

The researchers are skirting this issue by beginning work with another species, Roborovski dwarf hamsters (Phodopus roborovskii), which are smaller than mice and less aggressive than Syrian hamsters. The dwarf hamsters also get much sicker than their larger relatives following SARS-CoV-2 infection, developing massive blood clots in the lungs, which are often present in severe cases of COVID-19 in people as well. Most of these hamsters eventually die of the complications of the infection, which, according to Trimpert, indicates that they might be a good model of severe disease in humans.

A human intestinal organoid (with nuclei labeled in blue and cell shape outlined in green) infected with SARS-CoV-2 (labeled in red)

Joep Beumer

Getting a better picture of what is happening in people is a common goal. Eric Song, an MD/PhD student in Akiko Iwasakis lab at Yale School of Medicine, wanted to determine what effect SARS-CoV-2 was having on the brain. He and his colleagues used a humanized mouse model, in which a promoter drives the expression of human ACE2 in epithelial cells, in a study released as a preprint earlier this year. They found that SARS-CoV-2 could infect the central nervous system in the mice, but it still wasnt clear whether those findings translated to human patients.

To probe that question, the researchers turned to brain organoidscultured, miniature organs that resemble the developing brain and contain neuronal and glial cell types derived from human induced pluripotent stem cells (iPSCs). Not only did SARS-CoV-2 infect the organoids, infection also appeared to cause cell death, a finding corroborated by another study in brain organoids published by separate group September 23. Song and his colleagues also found antibodies against SARS-CoV-2 in the cerebrospinal fluid of people with COVID-19 and evidence of SARS-CoV-2 infection in the post-mortem brains of COVID-19 patients.

The team used three complementary methods to account for the limitations of each, Song tells The Scientist.There is room for all the different models that are in play, he adds. With human samples, you can only take really a snapshot of the sickest patients because thats the only time you will be getting a post-mortem sample, but combined with the mouse and the organoid model, youre able to study [the] kinetics and the dynamic process of viral invasion. Things like organoids really help us access tissues that we would not otherwise have access to.

Immunologist and developmental biologist Hans Clevers of Utrecht University in the Netherlands and colleagues are leveraging that access with gut organoids. Rather than deriving the organoids from iPSCs, though, they use multipotent stem cells taken from any adult epithelial tissue. In some ways the strategy is more limited: gut epithelial cells can only make mini guts, for instance, in contrast to iPSC-derived organoids, which can become lots of different tissues. On the other hand, they grow forever, Clevers says. iPSC-based organoids, you make them and then you have to use them for an experiment because they stop growing the moment you start specifying them.

His group published a study on July 3 investigating whether or not SARS-CoV-2 can target the gut, a hypothesis based on the expression of ACE2 in the intestinal lining and the gastrointestinal symptoms that many COVID-19 patients experience. Their work in human small intestinal organoids confirmed that the virus does enter the cells of the intestinal lining, replicate, and cause changes in gene expression.

Now, Clevers and his colleagues are using organoids to test possible COVID-19 therapeutics. Chloroquine is an example of a drug that looked promising for blocking SARS-CoV-2 infection when researchers tried it in conventional cell culturein that case, cells derived from African green monkey kidneys and grown in one layer on a dish, he says. But, as theyve shown in a new study thats been submitted for peer review, in the gut organoid model chloroquine is ineffective against SARS-CoV-2, just as it is in patients. According to Clevers, this latest finding indicates that an extra screening step in organoids following the identification of a promising drug in a traditional cell culture system could provide another layer of information before moving into preclinical animal models or the clinic.

Cell lines are cheap, they grow fast, and most robotic strategies for screening have been designed for cells growing in two dimensions, Clevers says. There are multiple efforts now around the world to come up with the machines to do similar high throughput screens with 3D. With the organoids, its a little bit more complex to analyze because theyre not flat, he explains, but the type of organoids they generate in his lab grow quickly. He predicts that organoids will come to be used more widely because their physiology recapitulates that of humans so well, but that theyll never replace animals.

Along with hamsters, nonhuman primates are one of the main models that researchers studying COVID-19 therapeutics use. SARS-CoV-2 readily infects primates, such as macaques, due to the compatibility of their ACE2 receptor, but they do not show disease symptoms, says Martinot. The nonhuman primate experience of COVID-19 seems to be most similar to the mildly symptomatic cases of most people who are infected, she adds. They develop regions of pathology in their lungs that are detectable with a microscope, but its not enough to make them sick, and observing that kind of disease in a person is unlikely because these animals are euthanized early in infection to allow researchers to track any changes.

It would be nice to have a primate model of severe disease, says Barouch. Nobody has been able to develop one so far, but models are always in development.

And symptom presentation isnt everything. These animals are harder to access and harder to house than rodents, but they are a wonderful model for vaccine research and for evaluating . . . the adaptive immune response to COVID-19, Martinot says, at least in part because there are so many existing tools researchers can leverage. Reagents that are available for evaluating the immune response [in people] work in monkeys, and so we can very carefully monitor the nonhuman primate for the development of antibodies, cytokine responses, and T-cell responses, she adds.

You always want to pick your best model for your specific question, Martinot tells The Scientist. But depending on what kind of question youre asking, or what kind of drug youre testing, you really have to choose the model that best fits your ability to answer those questions in a very accurate and reproducible way.

The World Health Organizations Research & Development Blueprint Team has been working on this since February. The team reviews progress and coordinates efforts on animal models of COVID-19 with the primary goal of advancing the development of COVID-19 therapeutics and vaccines. On September 23, some group members and a handful of other researchers published a review detailing the work thats been done to model SARS-CoV-2 infections in animalsparticularly, mice, hamsters, ferrets, and nonhuman primatesand highlighting how findings in animals correspond to disease progression in people.

The development and use of these models is not a linear process, and theyre under constant revision, says Barouch, who is part of the WHO working group. When there is human data, then that allows back validation or refinement of the model . . . so there has to be a continuous feedback from models to clinical trials and back.

In things that are so new, like SARS-CoV-2, it may well be that there is more than one useful animal model, Trimpert says. We should be open and flexible, especially in emergency situations like this.

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US FDA approves TaiGens IND application for influenza drug TG-1000 – CueReport

November 7th, 2020 5:51 am

TaiGen Biotechnology Company, a Taiwanese pharmaceutical company engaged in finding novel solutions to diabetes related complications, cancer, and infectious diseases, has reportedly announced that the U.S. FDA (Food and Drug Administration) has approved its IND (Investigation New Drug) application for TG-1000, a new treatment for both influenza B and A.

Along with TG-1000, TaiGen also has three more self-discovered NCEs: Furaprevir, an inhibitor of HCV protease to treat chronic hepatitis infection, TG-3000, an antagonist chemokine receptor used for chemosensitization and stem cell transplantation, and Taigexyn, a new non-fluorinated quinolone which is available in intravenous as well as oral formulations.

TaiGens novel pan influenza antiviral, TG-1000, stops viral transmission and replication using a cap-snatching mechanism. It can effectively work against influenza-B, influenza-A, Tamiflu-resistant viruses, and avian flu H7N9. The United States Patent and Trademark Office granted the first US patent for TG-1000 on 14th January 2020.

CEO and Chairman of TaiGen, Kuo-Lung Huang stated that influenza is a fatal disease with substantial unmet medical needs as well as heavy medical burden. Now, after the approval for IND by the U.S. FDA, the company is eager and ready to extend its clinical trials for TG-1000 in the United States over the coming years.

As per the Global Data, in 2019, the international market for influenza antivirals touched a valuation of $2.34 billion and is projected to hit the $5.03 billion valuation mark by the end of 2026, registering a CAGR of 11.5%.

Presently, the influenza antivirals industry only contains neuraminidase inhibitor oseltamivir and baloxavir, newly developed endonuclease inhibitor. With the recent launch of baloxavir in the industry, institutional investment analysts predict the endonuclease inhibitors' market share to rise at the cost of neuraminidase inhibitors. The companys TG-1000 is set take the complete advantage of this ongoing development.

Source credit: https://www.biospace.com/article/releases/taigen-announces-fda-approval-of-ind-for-its-flu-antiviral-tg-1000/#:~:text=TAIPEI%2C%20Taiwan%2C%20Nov.%201,for%20influenza%20A%20and%20B.

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Hunter Syndrome Treatment Market Size, Share, Market Research and Industry Forecast Report, 2026 (Includes Business Impact of COVID-19) – The Think…

November 7th, 2020 5:51 am

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Hunter Syndrome Treatment market.

Trusted Business Insights presents an updated and Latest Study on Hunter Syndrome Treatment Market. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Hunter Syndrome Treatment market during the forecast period.It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Hunter Syndrome Treatment Market Size, Share, Market Research and Industry Forecast Report, 2026 (Includes Business Impact of COVID-19)

Industry Insights, Market Size, CAGR, High-Level Analysis: Hunter Syndrome Treatment Market

The global Hunter syndrome treatment market size was valued at USD 864.9 million in 2018 and is expected to witness attractive growth over the forecast period. Introduction of novel therapies, robust product pipeline, rising government initiatives, and increasing awareness regarding Hunter syndrome and its available therapeutic options are expected to significantly fuel the market growth over the forecast period.Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder caused by an iduronate-2-sulfatase enzyme deficiency. Presently, there is no permanent cure for Hunter syndrome. Existing treatment including enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) focuses on providing symptomatic relief and management of complications associated with the disease progression.

According to the data published by the National Institute of Health (NIH) in 2018, Hunter syndrome affects around 1 in 160,000 males globally. The risk of developing this disease is far less among women because they inherit two X chromosomes and one of them can provide a functioning gene if the other X chromosome is defective. However, in men, there is no other X chromosome to compensate for the defective one.Expected approval of novel therapies in late-phase clinical trials and increasing R&D activities by key players for the development of such novel therapies are anticipated to be two major factors driving the market growth in the near future. For instance, in June 2019, Denali Therapeutics Inc. received FDAs Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation for its pipeline drug candidate DNL310, which is being evaluated for the treatment of the disease.Initiatives undertaken by various organizations for creating awareness regarding the disease diagnosis and its treatment is expected to support market growth over the forecast timeframe. For instance, in May 2018, Shire Plc. in collaboration with the National MPS Society and International MPS Network launched its third #FlyforMPS digital campaign aimed to increase awareness about .Treatment InsightsBased on treatment type, the market is categorized into enzyme replacement therapy (ERT), hematopoietic stem cell transplant (HSCT), and others. In 2018, the ERT segment accounted for the largest market share and is anticipated to hold onto its dominance over the forecast period. This is attributed to improved sales of Shire Plcs ELAPRASE and a potential for worldwide approval of GC Pharmas product called Hunterase.

Shire Plcs Elaprase (idursulfase) is the single major drug used for the treatment of Hunter syndrome, with GC Pharmas Hunterase (idursulfase beta) being approved only in South Korea as of now. These drugs have addressed a significant unmet need. However, the high cost of these drugs is expected to be a major factor hindering their market growth. For instance, Idursulfase (Elaprase) drug costs around USD 3,100 per 6mg/3ml vial.

Regional Insights of Hunter Syndrome Treatment Market

In 2018, North America dominated the market owing to favorable regulations for orphan drug development, rising awareness among people concerning rare diseases, increased funding for research activities, and improved healthcare facilities. Furthermore, the favorable reimbursement policies for expensive drugs such as ELAPRASE in the U.S. have supported its adoption and fueled the regional growth.Asia Pacific region is projected to exhibit a lucrative growth rate over the forecast period. Japan, China, and India are expected to emerge as potential countries for growth, owing to their high unmet needs in the market. Major players are focused on gaining approval for their novel therapies and are penetrating these markets to attain a major share. For instance, in July 2019, CANBridge Pharmaceuticals Inc. filed a New Drug Application (NDA) with the National Medical Products Administration (NMPA) for its novel treatment drug called Hunterase in China. Hunterase (idursulfase beta) is a patented therapy of GC Pharma indicated for the treatment of Hunter syndrome.

Market Share Insights of Hunter Syndrome Treatment Market

Some of the key players in the market comprise Shire Plc. (Takeda Pharmaceutical Company); GC Pharma; JCR Pharmaceuticals Co Ltd.; RegenxBio Inc.; Sangamo Therapeutics, Inc.; ArmaGen Inc; Inventiva S.A.; Denali Therapeutics Inc.; Bioasis Technologies Inc.; and Esteve.Currently, Shire Plc. (acquired by Takeda Pharmaceutical Company Limited in April 2019) is a prominent market player, with strong sales of its drug ELAPRASE, indicated for the treatment of Hunter syndrome. However, Shire plc is expected to face stiff competition from Green Cross (GC) Pharma over the forecast period. GC Pharma is emerging as a global player in the Hunter syndrome treatment market with its orphan drug, Hunterase.GC Pharma is undertaking inorganic growth strategies such as partnerships and collaborations for the commercialization and geographical expansion of Hunterase. For instance, in April 2019, Clinigen Group plc and GC Pharma entered into an exclusive licensing agreement under which Clinigen gained the rights to commercialize Hunterase in Japan.

Segmentations, Sub Segmentations, CAGR, & High-Level Analysis overview of Hunter Syndrome Treatment Market Research ReportThis report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest trends and opportunities in each of the sub-segments from 2015 to 2026. For the purpose of this study, this market research report has segmented the global Hunter syndrome treatment market report on the basis of treatment and region:

Treatment Outlook (Revenue, USD Million, 2019 2030)

Enzyme Replacement Therapy (ERT)

Hematopoietic Stem Cell Transplant (HSCT)

Others

Looking for more? Check out our repository for all available reports on Hunter Syndrome Treatment in related sectors.

Quick Read Table of Contents of this Report @ Hunter Syndrome Treatment Market Size, Share, Market Research and Industry Forecast Report, 2026 (Includes Business Impact of COVID-19)

Trusted Business InsightsShelly ArnoldMedia & Marketing ExecutiveEmail Me For Any ClarificationsConnect on LinkedInClick to follow Trusted Business Insights LinkedIn for Market Data and Updates.US: +1 646 568 9797UK: +44 330 808 0580

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[Joint optic disc and cup segmentation based on residual multi-scale fully convolutional neural network]. – Physician’s Weekly

November 5th, 2020 9:56 am

Glaucoma is the leading cause of irreversible blindness, but its early symptoms are not obvious and are easily overlooked, so early screening for glaucoma is particularly important. The cup to disc ratio is an important indicator for clinical glaucoma screening, and accurate segmentation of the optic cup and disc is the key to calculating the cup to disc ratio. In this paper, a full convolutional neural network with residual multi-scale convolution module was proposed for the optic cup and disc segmentation. First, the fundus image was contrast enhanced and polar transformation was introduced. Subsequently, W-Net was used as the backbone network, which replaced the standard convolution unit with the residual multi-scale full convolution module, the input port was added to the image pyramid to construct the multi-scale input, and the side output layer was used as the early classifier to generate the local prediction output. Finally, a new multi-tag loss function was proposed to guide network segmentation. The mean intersection over union of the optic cup and disc segmentation in the REFUGE dataset was 0.904 0 and 0.955 3 respectively, and the overlapping error was 0.178 0 and 0.066 5 respectively. The results show that this method not only realizes the joint segmentation of cup and disc, but also improves the segmentation accuracy effectively, which could be helpful for the promotion of large-scale early glaucoma screening.

PubMed

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[Joint optic disc and cup segmentation based on residual multi-scale fully convolutional neural network]. - Physician's Weekly

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Six great shows to get your culture fix in Toronto this month – Post City

November 5th, 2020 9:56 am

Digital theatre, live environment

The Princess of Wales Theatre is scheduled to present the first indoor theatre show in Toronto since the pandemic shutdown. Scheduled to open Nov. 17, Mirvish will be presenting the international premiere of the Donmar Warehouse production of Blindness, a socially distanced sound installation. The date is subject to change based on health and safety protocols.

Our friends at the Donmar Warehouse in London came up with a brilliant and powerful way to tell a timely and important story in a theatrical setting. They came up with a unique adaptation of the modern classic novel Blindness by the Nobel laureate Jos Saramago, itself about the effects of a pandemic on a community, says David Mirvish. Blindness was a resounding success in August and September at the Donmar and helped to usher in the return of theatrical events.

The show runs 70 minutes with no intermission, and there will only be 50 people in attendance per performance. According to Mirvish, the sound is designed to be binaural, making the work sound as if it were physically happening around you, putting you in the centre of the action.

Two new documentary photography exhibitions opening at the Art Gallery of Ontario give Torontonians their first chance to have a look at some of the latest acquisitions of works by African-American artists, including Dawoud Bey, John Edmonds, Wardell Milan and Ming Smith, as well as Malian photographer Malick Sidib. Its part of the gallerys commitment to greater diversity and representation.

The Documents, 1960s1970s exhibition focuses attention on a moment of great social change internationally and features both studio portraits and street scenes.

Opening on the same day in the AGOs Robert & Cheryl McEwen Gallery, the exhibition Dawoud Bey, John Edmonds and Wardell Milan features works by three contemporary African-American artists exploring and re-envisioning the history of Black representation and the Black American experience.

Acclaimed singer-songwriter Donovan Woods will be live-streaming a one-hour solo acoustic concert at Roy Thomson Hall on Nov. 5. The performance, which was recorded by seven robotic cameras while Woods was alone on stage in the Toronto concert hall, is in celebration of his seventh studio album release, Without People.

In an effort to support others with the release of his album, 50 per cent of net ticket sales will be given back to participating show promoters, and Woods will donate 100 per cent of his artist proceeds to ArtsCan Circle (Canada) and Southern Girls Rock Camp (US). During the show, Woods will perform a nine-song set of songs off the new album and a few fan favourites. Philadelphia-based group Lullanas will open the show. Tickets are $15.

The Factory Theatre is kicking off its new season with a live streaming world premiere of acts of faith by award-winning Asian Canadian playwright David Yee, directed by Nina Lee Aquino, and starring Natasha Mumba. According to the Factory, this production has been written specifically to be performed in a digital environment.

The story revolves around the main character, named Faith, who is mistaken for a prophet. Acts of faith is a story about the power of faith, the inescapable persistence of our online identities and the nature of truth in a digital age. Sounds timely.

Acts of faith will stream live to audiences at home for six performances, Nov. 1928. Admission is free of charge.

The Kensington Market Jazz Festival is offering two days of incredible live stream performances Nov. 78. One of those headlining the weekend of performances is jazz pianist and vocalist Champian Fulton, who is described as one of the most gifted and pure jazz musicians of her generation. She will be playing, alongside saxophonist Nick Hempton, Sunday evening, Nov. 8, at 9:30 p.m. Other performers include Paul Marinaro, Carol Welsman, Jane Bunnett and many more.

The Musical Stage Company will be offering a virtual edition of its signature concert event, Uncovered: Notes From the Heart, running from Nov. 11 to Dec. 6 and featuring some of the citys top musical theatre performers, such as Divine Brown, fresh off a run on Broadway where she performed in Harry Potter and the Cursed Child, as well as Hailey Gillis, Bruce Dow and others.

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The invisible breast cancer: Experience does not protect against inattentional blindness to clinically relevant findings in radiology. – Physician’s…

November 5th, 2020 9:56 am

Retrospectively obvious events are frequently missed when attention is engaged in another task-a phenomenon known as inattentional blindness. Although the task characteristics that predict inattentional blindness rates are relatively well understood, the observer characteristics that predict inattentional blindness rates are largely unknown. Previously, expert radiologists showed a surprising rate of inattentional blindness to a gorilla photoshopped into a CT scan during lung-cancer screening. However, inattentional blindness rates were higher for a group of nave observers performing the same task, suggesting that perceptual expertise may provide protection against inattentional blindness. Here, we tested whether expertise in radiology predicts inattentional blindness rates for unexpected abnormalities that were clinically relevant. Fifty radiologists evaluated CT scans for lung cancer. The final case contained a large (9.1 cm) breast mass and lymphadenopathy. When their attention was focused on searching for lung nodules, 66% of radiologists did not detect breast cancer and 30% did not detect lymphadenopathy. In contrast, only 3% and 10% of radiologists (N = 30), respectively, missed these abnormalities in a follow-up study when searching for a broader range of abnormalities. Neither experience, primary task performance, nor search behavior predicted which radiologists missed the unexpected abnormalities. These findings suggest perceptual expertise does not protect against inattentional blindness, even for unexpected stimuli that are within the domain of expertise.

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Genetic Testing Can Lead to Precision Medicine Therapies for Cancer – HealthITAnalytics.com

November 5th, 2020 9:55 am

November 02, 2020 -Broad-based genetic testing could identify inherited genetic mutations and accelerate precision medicine therapies for patients with cancer, according to a study published in JAMA Oncology.

Hereditary factors play a key role in the development of many cancers, researchers at Mayo Clinics Center for Individualized Medicine noted. Identifying genetic predispositions for certain cancers can have significant implications for treatment decisions, interventions, cancer screenings, and genetic testing for patients and close relatives.

Selecting patients for genetic testing has traditionally been based on pathologic features of the cancer, age at diagnosis, family history of cancer, and other factors named in clinical guidelines. Few studies have examined the impact of broad-based testing for gene mutations in patients with cancer compared with more traditional methods of selection.

Over the course of two years, Mayo Clinic researchers provided free genetic testing and counseling for 3,084 patients as part of their standard cancer care. The project included a wide range of cancer stages and types, such as breast, colorectal, lung, ovarian, pancreatic, bladder, and prostate cancers.

The results showed that with standard guidelines, physicians were only able to find 48 percent of patients with an inherited genetic mutation.

We found that 13.5 percent of patients had an inherited mutation in a gene associated with the development of their cancer, saidNiloy Jewel Samadder, MD, a Mayo Clinic gastroenterologist and hepatologist, who is the study's author.

More than half of the patients who developed cancer due to inherited mutations were being missed, and that has major implications for family members. Everyone has some risk of developing cancer, and in most cases the disease develops by chance. However, some people are genetically predisposed to developing certain types of cancer, such as breast or colon cancers.

When researchers examined the effects of a genetic mutation discovery, the team found that one-third of patients with the highest-risk cancer genes had a change in their medical management, including the type of surgery or chemotherapy they received.

This targeted treatment would have been lost if the patients had not received genetic testing, Samadder stated.

The results demonstrate the importance of genetic testing for all patients, and not just specific individuals.

Genetic testing is underutilized in cancer care, both for patients and for their families, often due to outdated guidelines that restrict testing to a narrow group of high-risk patients, saidRobert Nussbaum, MD,chief medical officer ofInvitae Corporation.

All cancer patients should have access to complete genetic information that can guide their care and inform their families' health.

Additionally, high-risk patients should share the heritable-cause of the disease with their relatives, which will allow family members to pursue disease care for earlier cancer management and detection.

We can help prevent cancer in their loved ones because it is genetic, and they share these cancer-causing genetic changes with their children, siblings and others in their families, Samadder said. We can target prevention strategies for those high-risk individuals and hopefully prevent cancer altogether in future generations of their family.

All blood-related family members of patients found to have a genetic mutation were offered free genetic testing. Overall, one in five of these family members underwent testing, the researchers said.

Going forward, the research team hopes to be able to incorporate the studys results into the care of all patients with cancer at Mayo Clinic. The study demonstrates the potential for broad genetic testing to accelerate the development of precision medicine therapies for cancer.

Steps are being taken to ensure all patients are offered genomic sequencing to better understand the genes that led to the development of their cancer, and how to precisely target treatment and improve survival, said Samadder.

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Modalis Therapeutics Reports Third Quarter 2020 Financial Results and Operational Highlights – BioSpace

November 5th, 2020 9:55 am

Nov. 5, 2020 06:00 UTC

TOKYO & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Modalis Therapeutics Corporation (Modalis) (TOKYO: 4883), a leading company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM epigenetic gene modulation technology, today reported financial results for the third quarter ended September 30, 2020, as well as recent operational highlights.

"Our goal is to create CRISPR based gene therapies for genetic disorders, most of which fall into the orphan disease category. There should be no disease that is ignored because of its small patient population, and our mission to develop disease modifying treatments for these diseases reflects our belief that Every Life Deserves Attention. We are proud to be a pioneer in CRISPR based gene modulation therapies and we are grateful to our investors and employees who are working to fulfill this important mission, said Haru Morita, Chief Executive Officer of Modalis.

Operational Highlights:

Third Quarter 2020 Financial Results:

About Modalis:

Modalis Therapeutics is developing precision genetic medicines through epigenetic gene modulation. Founded by Osamu Nureki and leading scientists in CRISPR gene editing from University of Tokyo, Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or histone modification without the need for double-stranded DNA cleavage, gene editing or base editing. Modalis is focusing initially on genetic disorders caused by loss of gene regulation resulting in excess or insufficient protein production which includes more than 660 genes that are currently estimated to cause human disease due to haploinsufficiency. Headquartered in Tokyo with laboratories and facilities in Cambridge, Massachusetts. For additional information, visit http://www.modalistx.com.

Consolidated Financial Results for the Nine Months Ended September 30, 2020 [Japanese GAAP]

Company name: Modalis Therapeutics CorporationStock exchange listing: Tokyo Stock ExchangeCode number: 4883URL: https://www.modalistx.com/jp/ Representative: Haruhiko Morita, President and Representative DirectorContact: Naoki Kobayashi, CFO and Executive OfficerPhone: +81-3-6822-4584Scheduled date of filing quarterly securities report: November 13, 2020Scheduled date of commencing dividend payments: -Availability of supplementary briefing material on quarterly financial results: AvailableSchedule of quarterly financial results briefing session: -

(Amounts of less than one million yen are rounded down.)

1.

Consolidated Financial Results for the Nine Months Ended September 30, 2020 (January 1, 2020 to September 30, 2020)

(1) Consolidated Operating Results

(% indicates changes from the previous corresponding period.)

Operating revenue

Operating income

Ordinary income

Profit attributable toowners of parent

Nine months ended

Million yen

%

Million yen

%

Million yen

%

Million yen

%

September 30, 2020

340

-

168

-

209

-

214

-

September 30, 2019

-

-

-

-

-

-

-

-

(Note)

Comprehensive income:

Nine months ended September 30, 2020: 215 million [-%]

Nine months ended September 30, 2019: - million [-%]

Basic earnings

per share

Diluted earnings

per share

Nine months ended

Yen

Yen

September 30, 2020

8.34

-

September 30, 2019

-

-

(Notes)

1. The Company has not prepared the consolidated financial statements for the nine months ended September 2019. Accordingly, no figures are shown for the nine months ended September 30, 2019 and no percentage changes are shown for the nine months ended September 30, 2020.

2. Although the Company has dilutive shares, diluted earnings per share are not indicated because the Companys shares were not listed and the average share price is not available for the period under review.

(2) Consolidated Financial Position

Total assets

Net assets

Capital adequacyratio

Million yen

Million yen

%

As of September 30, 2020

6,480

6,428

99.2

As of December 31, 2019

3,938

3,842

97.6

(Reference)

Equity:

As of September 30, 2020: 6,428 million

As of December 31, 2019: 3,842 million

View source version on businesswire.com: https://www.businesswire.com/news/home/20201104005831/en/

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Biocryst treatment for genetic disease HAE lands early access approval in UK – WRAL Tech Wire

November 5th, 2020 9:55 am

RESEARCH TRIANGLE PARK The United Kingdoms Medicines and Healthcare products Regulatory Agency (MHRA) has granted Durham-basedBioCryst Pharmaceuticals berotralstat a positive scientific opinion through the Early Access to Medicines Scheme.

Berotralstat is an oral, once-daily medication for hereditary angioedema (HAE), a serious and potentially life-threatening, rare genetic illness characterized by periodic episodes of acute swelling in various parts of the body including skin, throat, gastrointestinal tract and extremities.

With the regulatory agencys positive opinion, hereditary angioedema patients in the U.K. aged 12 years and older can gain access to berotralstat for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the European Commission.

HAE patients around the world are waiting for an oral, once-daily therapy to prevent attacks and reduce their burden of therapy, said Jon Stonehouse, chief executive officer of BioCryst. With this decision by the MHRA, the wait for many HAE patients in the U.K. can end sooner.

Durhams BioCryst lands $44M contract to test anti-viral drug against COVID-19 virus

Medicines included in the United Kingdoms Early Access to Medicines Scheme (EAMS) are those that have a high unmet need, are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where there are no adequate treatment options, and are likely to offer significant advantage over methods currently used in the country. Under the scheme, the MHRA provides a scientific opinion on the benefit-risk balance of the medicine, based on the data available when the EAMS submission was made.

There are many patients in the U.K. that dont have a realistic option for effective HAE prophylaxis, said Dr. Sorena Kiani, consultant immunologist at Royal London Hospital, London. The addition of berotralstat through the EAMS will bring a much needed option for HAE patients suffering with this debilitating disease.

The European Medicines Agency is reviewing the marketing authorization application for berotralstat under the centralized procedure. An opinion from the Committee for Medicinal Products for Human Use is expected approximately 12 months from the marketing authorization application validation, which the company announced on March 30, 2020.

BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease.

(C) N.C. Biotech Center

RTP biotech BioCryst receives $7M order from feds for influenza therapy

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Moms of kids with autism may show subtle signs of the condition themselves – Insider – INSIDER

November 5th, 2020 9:55 am

The mothers of kids with autism often have subtle traits of the condition that are not enough for an autism diagnosis, but that could indicate a genetic link for autism spectrum disorder, according to a new study.

The study, published in the journal Biological Psychiatry, looked at genetic and behavioral information from 2,614 families in which one child has autism. The researchers found that women who have trouble communicating in social settings tend to have children with autism who have more pronounced social and communication challenges, according to Spectrum News.

The findings could help researchers better understand the role of genetics in autism spectrum disorder, and how the condition presents in women. With autism becoming increasingly common affecting 1 in 59 American children a better understanding of the condition is critical.

The researchers looked at parents of children with autism using the broad autism phenotype (BAP). A phenotype is the way that genetic information is expressed. Researchers theorize that BAP includes subtle signs of the symptoms associated with autism spectrum disorder. For example, a person might be sensitive to sensory stimulations or have some trouble communicating, but not enough to warrant an autism diagnosis.

Understanding how common BAP is in the families of people with autism can help researchers understand how likely it is that someone with a genetic predisposition to autism will develop the condition. This is also known as genetic liability.

"I was really excited to see that features of broad autism phenotype, and especially language-related features, seem to be really important in understanding how genetic liability is expressed and really linked to molecular genetic variation," Molly Losh, director of the Neurodevelopmental Disabilities Lab at Northwestern University in Evanston, Illinois and lead author of the study, told Spectrum News.

The study indicated that women with BAP could pass down a genetic predisposition to autism, even when they don't have the condition themselves. This is known as the female protective effect the idea that it takes more genetic influence to lead to autism in females than males. That could be why autism is diagnosed more often in boys.

The researchers evaluated both mothers and fathers for BAP,using a questionnaire. Overall, dads had a higher BAP score or more tendencies that could be associated with BAP.

The researchers found that mothers and fathers BAP scores were linked to the behaviors of their children with autism in different ways. If dads had a rigid approach to the world, their children were more likely to have repetitive behaviors. If moms had a high BAP score, their children with autism were more likely to have symptoms related to language, communication, and social cues.

This study is significant because while previous research has looked at the correlation between fathers' BAP scores and their children's symptoms, this is the first study to link mother's BAP scores with children's symptoms.

Losh and Lea Davis, assistant professor of genetic medicine at Vanderbilt University in Nashville, Tennessee, and co-author of the study, plan to do more research into female BAP and how that plays out in families where children have autism. That could help researchers understand more about how autism presents in females.

"The field is really good at identifying these features at a granular level for young boys, really not nearly as good at doing that for, let's say, adult women," Davis told Spectrum News. "That's another area that we're just starting to scratch the surface on, and this was an interesting way of kind of looking at some of those questions."

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Investigating the genetic characteristics of CAD: Is there a role for myocardial perfusion imaging techniques? – Physician’s Weekly

November 5th, 2020 9:55 am

Several environmental and genetic factors have been found to influence the development and progression of coronary artery disease (CAD). Although the effects of the environmental hazards on CAD pathophysiology are well documented, the genetic architecture of the disease remains quite unclear. A number of single-nucleotide polymorphisms have been identified based on the results of the genome-wide association studies. However, there is a lack of strong evidence regarding molecular causality. The minority of the reported predisposing variants can be related to the conventional risk factors of CAD, while most of the polymorphisms occur in non-protein-coding regions of the DNA. However, independently of the specific underlying mechanisms, genetic information could lead to the identification of a population at higher genetic risk for the long-term development of CAD. Myocardial single-photon emission computed tomography (SPECT) and positron emission tomography (PET) are functional imaging techniques that can evaluate directly myocardial perfusion, and detect vascular and/or endothelial dysfunction. Therefore, these techniques could have a role in the investigation of the underlying mechanisms associated with the identified predisposing variants, advancing our understanding regarding molecular causality. In the population at higher genetic risk, myocardial SPECT or PET could provide important evidence through the early depiction of sub-clinical dysfunctions, well before any atherosclerosis marker could be identified. Notably, SPECT and PET techniques have been already used for the investigation of the functional consequences of several CAD-related polymorphisms, as well as the response to certain treatments (statins). Furthermore, therefore, in the clinical setting, the combination of genetic evidence with the findings of myocardial SPECT, or PET, functional imaging techniques could lead to more efficient screening methods and may improve decision making with regard to the diagnostic investigation and patients management.

PubMed

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Nancy Carrasco elected to the National Academy of Medicine for outstanding professional achievement and commitment to service – Vanderbilt University…

November 5th, 2020 9:55 am

On Oct. 19,Nancy Carrasco, professor and chair of the Department of Molecular Physiology and Biophysics and the Joe C. Davis Chair of Biomedical Science, waselectedto the National Academy of Medicine.

The election process recognizes individuals who have made major contributions to the advancement of the medical sciences, health care and public health. According to a release, current members elected Carrasco for making exceptional contributions to elucidating mechanisms by which ions and other solutes are transported across biological membranes. Her work has broad impact and significance across biomedical fields ranging from biophysics and molecular physiology to cancer, metabolism, molecular endocrinology, and public health.

We are thrilled that Dr. Carrasco has been recognized by the National Academy of Medicine for the work that she continues to devote her extraordinary career to, saidLawrence Marnett, dean of the Vanderbilt University School of Medicine Basic Sciences and Mary GeddesStahlmanProfessor of Cancer Research. Her research is focused on understanding the physiology of thyroid hormone biosynthesis and how it is affected by genetic mutations and environmental pollutants. She is addressing pressing public health concerns, and her work has a clear, tangible impact on human health.

Dr. Carrascos election to the National Academy of Medicine underscores her commitment to bringing scientific clarity to a public health crisis. Her focus on inclusive and collaborative research has resulted in transformative research that is meaningfully improving human health, while also exemplifying the diverse perspectives and trans-institutional methods that set Vanderbilt apart, noted Provost and Vice Chancellor for Academic AffairsSusan R. Wente.

Carrasco has been elected to the NAM along withtwo other Vanderbilt researchers,Velma McBride Murry, university professor of health policy and human and organizational development in Peabody College and the School of Medicine and the Lois Autrey Betts Chair of Education and Human Development at Peabody College, andConsuelo Wilkins, professor of medicine in the School of Medicine and vice president for health equity at Vanderbilt University Medical Center.

Carrasco isolated the coding DNA for the sodium/iodide symporter NIS, the iodide transporter protein that actively pulls iodide from the bloodstream into the thyroid gland. Iodide is an essential constituent of the thyroid hormones, which are crucial for the development of the nervous system beginning in uterine life, and regulate metabolism in virtually all tissues. The critical importance of the thyroid hormones makes understanding the protein that ushers their key constituent into the thyroid gland essential to understanding human health overall.

I am deeply honored to have been elected to the National Academy of Medicine, Carrasco said. I have always felt very strongly that the links between understanding physiology and pathophysiology at the molecular level and both medical practice and public health should be viewed as a cornerstone of our collective efforts to improve the health of our communities, and that has been a guiding principle in my work. I am extremely grateful to the members of the Academy for electing me and, in so doing, affirming the value of basic science as a key contributor to progress in medicine.

Carrasco continues to investigate the functions of NIS and its interaction with the environmental pollutantperchlorate. She and her colleagues recently reported that perchlorate exposure fundamentally alters the mechanism by which NIS transports iodide into the thyroid, and her group had previously shown that NIS is functionally expressed in lactating breast tissue, making it clear that this pollutant is more dangerous than previously thought. These discoveries demonstrate that perchlorate exposure can markedly decrease thyroid hormone production in vulnerable populations, including pregnant and nursing mothers and their fetuses and newborns. Her research also has direct applications to the development of breast cancer therapeutics.

Carrasco has received numerous national and international awards, including the Pew Award in the Biomedical Sciences, the Arnold and Mabel Beckman Foundation Award, the Maria SibyllaMerianAward (Germany), the Merck Prize from the European Thyroid Association (Poland), the NounShavitAwardin Life Sciences (Israel),and Light of Life Award. She has served as president of the Society of Latin American Biophysicists and was elected to the National Academy of Sciences in 2015.

Carrasco received her M.D. and masters degree in biochemistry from the National Autonomous University of Mexico in her native Mexico City and completed her postdoctoral training at the Roche Institute of Molecular Biology. She joined the faculty at Albert Einstein College of Medicine in 1987 and at the Yale School of Medicine in 2011. She joined Vanderbilt in 2019.

This distinguished and diverse class of new members is a truly exceptional group of scholars and leaders whose expertise in science, medicine, health, and policy will be integral to helping the NAM address todays most pressing health challenges and inform the future of health and health care for the benefit of everyone around the globe, said National Academy of Medicine PresidentVictor J. Dzau. It is my privilege to welcome these esteemed individuals to the National Academy of Medicine.

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LogicBio Therapeutics Announces Appointment of Veteran Biotech Executive Mariana Nacht, Ph.D., as Chief Scientific Officer and Kyle Chiang, Ph.D.,…

November 5th, 2020 9:55 am

LEXINGTON, Mass., Nov. 03, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today announced the appointment of Mariana Nacht, Ph.D., as chief scientific officer, effective Nov. 30, 2020, and the promotion of Kyle Chiang, Ph.D., to chief operating officer, effective Nov. 2, 2020.

Dr. Nacht brings more than 20 years of experience in both large and small biotech companies to her role at LogicBio. Most recently, she served as CSO and was a founding executive team member of Cereius, where she led a small internal research team and a group of collaborators to develop radiolabeled proteins for the treatment of brain metastases. Before that, she served as CSO of Vivid Biosciences, a functional precision medicine company, where she was also a founding executive team member. Dr. Nacht has also served in key scientific roles at Padlock Therapeutics (acquired by Bristol Myer Squibb in 2014) and Avila Therapeutics, a platform company that developed covalent irreversible inhibitors and was acquired by Celgene in 2012. Earlier in her career, she spent a decade working at Genzyme (now Sanofi Genzyme), where she led anti-angiogenesis and oncology target discovery efforts. Dr. Nacht received her B.S. in biology from Tufts University and her Ph.D. from the University of Pennsylvania.

We are proud to expand our leadership team as we prepare to launch our first clinical trial in pediatric patients with methylmalonic acidemia (MMA) and continue to advance both our GeneRide and Next Generation Capsid platforms, said Fred Chereau, CEO of LogicBio. Mariana brings terrific expertise in novel therapeutic platforms as well as deep experience in building and leading strong scientific teams to her position as CSO. Were thrilled to welcome her to LogicBio as we move into this exciting next phase of progress. Im also delighted to have Kyle promoted to our core leadership team. He has provided essential guidance on pipeline strategy and program development from the early days of LogicBio and he will continue to be an important voice in shaping our future growth.

I am very enthusiastic about the potential for the GeneRide platform to transform care for pediatric patients with rare genetic diseases, Dr. Nacht said. We all enter this field to make a difference for patients, and I am excited to be joining LogicBio just as LB-001, our lead program for children with MMA, is about to enter the clinic with the Phase 1/2 SUNRISE trial. Beyond LB-001, I look forward to further advancing LogicBios pipeline with the goal of bringing more durable and transformational therapies to people living with devastating genetic diseases.

Dr. Chiang was the second employee at LogicBio and has held positions of increasing responsibility since joining the team as director of translational science in 2016. Most recently, he served as vice president, product strategy, where he led LB-001 through early regulatory interactions and managed LogicBios collaboration with the Childrens Medical Research Institute to develop more potent and more easily manufacturable AAV capsids for gene therapy and genome editing applications. Before joining LogicBio, Dr. Chiang led aTyr Pharmas ATYR1940 program from discovery through early clinical development for patients with facioscapulohumeral muscular dystrophy. Dr. Chiang received his B.S. in biochemistry and cell biology from the University of California, San Diego and his Ph.D. in macromolecular cellular structure and chemistry from the Scripps Research Institute.

LogicBio also announced today that Bryan Yoon, Esq., the companys chief administrative officer, general counsel and corporate secretary, will be departing from the company effective Nov. 6, 2020. I want to thank Bryan for his contributions to LogicBio and we wish him the best in his next challenge, Mr. Chereau said.

AboutLogicBioTherapeuticsLogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration withTakedato research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered in Lexington, Mass. For more information, please visit http://www.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the timing, progress and results of the Companys strategic directives and its research and development activities, including those related to LB-001 and its pipeline. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Investors:Matthew LaneGilmartin Investor Relationmatt@gilmartinir.com

Media:Stephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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Tests Show Genetic Signature of Coronavirus That Likely Infected Trump – The New York Times

November 5th, 2020 9:55 am

President Trumps illness from a coronavirus infection last month was the most significant health crisis for a sitting president in nearly 40 years. Yet little remains known about how the virus arrived at the White House and how it spread.

The administration did not take basic steps to track the outbreak, limiting contact tracing, keeping cases a secret and cutting out the Centers for Disease Control and Prevention. The origin of the infections, a spokesman said, was unknowable.

But one standard public health technique may still shed some light: tracking the clusters genetic fingerprints.

To better understand the outbreak, The New York Times worked with prominent geneticists to determine the genetic sequence of viruses that infected two Times journalists believed to have been exposed to the coronavirus as part of their work covering the White House.

The study reveals, for the first time, the genetic sequence of the virus that may have infected Mr. Trump and dozens of others, researchers said. That genome is a crucial clue that may allow researchers to identify where the outbreak originated and whether it went on to infect others across the country.

The White House has not disclosed any effort to conduct similar genetic testing, but the studys results show that it is still possible, even weeks after positive tests. Additional sequencing could help establish the path of the virus through the White House, the role of a possible super-spreading event for Judge Amy Coney Barrett and the origin of an outbreak among the staff of Vice President Mike Pence in the last week or so.

The journalists, Michael D. Shear and Al Drago, both had significant, separate exposure to White House officials in late September, several days before they developed symptoms. They did not spend any time near each other in the weeks before their positive tests.

Mr. Shear traveled with Mr. Trump and other staff on Air Force One on Sept. 26, when Mr. Trump approached within five or six feet without a mask. Mr. Drago covered the Judge Barrett event that day and a news conference the next day near officials who were not wearing masks and later tested positive. Both journalists wore masks.

The viral genomes of the two journalists shared the same distinct pattern of mutations, the research found. Along with their exposure history, the findings suggest that they were infected as part of the broader White House outbreak, said Trevor Bedford, a geneticist at the Fred Hutchinson Cancer Research Center and the University of Washington who led the research team.

These mutations that are possessed by these viruses are quite rare in the United States, Dr. Bedford said. I am highly convinced that these viruses come from the same outbreak or cluster based on their genomes.

The study, which has been posted online but not yet peer reviewed or published in a science journal, followed academic protocols that require genetic samples to be anonymous. Mr. Shear and Mr. Drago chose to disclose their identities for this article.

Viruses constantly mutate, picking up tiny, accidental alterations to their genetic material as they reproduce. Few mutations alter how a virus functions. But by comparing patterns of mutations across many genetic sequences, scientists can construct family trees of a virus, illuminating how it spreads.

The genomes believed by these researchers to be connected to the White House outbreak do not identify a recent geographic source, in part because they are unusual. The ancestors of those viruses spread to the United States from Europe and were circulating widely across the country in April and May, but the trail goes cold after that, according to Dr. Bedford.

Geneticists said the genomes are a key piece of the puzzle that may spur future research to determine where the White House outbreak originated and where it may go next. Scientists collect and publish tens of thousands of new sequences of the coronavirus every month, and additional testing may fill in the picture.

The results show that even weeks after it was identified, the White House outbreak would be better understood by sequencing samples of more people who were infected. Swabs used in positive tests are often kept in labs for months after an initial infection, and genetic material for the coronavirus is stable if stored appropriately.

The C.D.C. routinely relies on genetic testing to help understand Covid-19 outbreaks elsewhere across the country. In a study released on Thursday, the C.D.C. cited genetic sequencing and intensive contact tracing that documented an super-spreading event at a high school retreat in Wisconsin.

But the Trump administration is not known to have conducted its own genetic analysis of people infected in the outbreak. The White House declined to respond to questions on genetic sequencing of Mr. Trump and the cluster of aides and officials who tested positive or became ill.

There is still a remote possibility, Dr. Bedford said, that a previously unseen version of the virus had been circulating undetected in Washington or Northern Virginia and infected both journalists independently from the White House cluster. More testing of the outbreak could eliminate that possibility entirely, he said.

Scientists not involved in the research who reviewed the results agreed with the conclusion that the two samples sharing rare mutations strongly suggested they are part of the same outbreak.

These genomes are probably going to be identical or nearly identical to the genome that infected the president, said Michael Worobey, head of the department of ecology and evolutionary biology at the University of Arizona.

Dr. Worobey disputed the White Houses characterization that the source of the outbreak could not be known.

A lot of things are unknowable if you make no effort to know anything about them, and this falls into this category, Dr. Worobey said. All of these things actually can be known if you make the effort and you have the transparency that scientists are desperately trying to promote as we sequence hundreds of thousands of these genomes around the world.

For months, the White House minimized the threat of the virus and eschewed basic safety precautions at official events, like wearing a mask or keeping people six feet apart.

At least 11 people who attended a Rose Garden celebration on Sept. 26 for Judge Barrett, which included an indoor event without masks, became infected with the coronavirus, including Mr. Trump. Additional genetic testing could help more clearly establish the role of that event.

Dr. Bedford and his colleagues were able to obtain a full genetic sequence for the virus that infected Mr. Shear and a partial sequence of the virus that infected Mr. Drago. Several unusual mutations matched in the two samples, sufficient evidence to determine with a very high probability that they were essentially the same genome, Dr. Bedford said.

The work was carried out by a multidisciplinary team of researchers at the University of Washington School of Medicine, the Hutchinson Center and the Brotman Baty Institute for Precision Medicine in Seattle.

The work is convincing, and it is the best way to piece together the progression of such an outbreak, said David Engelthaler, head of the infectious disease branch of the Translational Genomics Research Institute in Arizona, where he and colleagues have sequenced thousands of genomes to track the spread of the coronavirus, including devastating outbreaks at Native American reservations in the state.

Its critical no matter where we are to sequence this virus, Dr. Engelthaler said. Not just at the White House, but at the White Mountain Apache Reservation here in Arizona.

Carl Zimmer contributed reporting.

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Tests Show Genetic Signature of Coronavirus That Likely Infected Trump - The New York Times

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Sarepta Therapeutics to Present at the 29th Annual Credit Suisse Virtual Healthcare Conference – GlobeNewswire

November 5th, 2020 9:55 am

CAMBRIDGE, Mass., Nov. 02, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in a fireside chat at the 29th Annual Credit Suisse Virtual Healthcare Conference on Monday, November 9, 2020 at 3:30 p.m. E.T.

The presentation will be webcast live under the investor relations section of Sareptas website at http://www.sarepta.com and will be archived there following the presentation for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the 'Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.Investors:Ian Estepan, 617-274-4052, iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

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Sarepta Therapeutics to Present at the 29th Annual Credit Suisse Virtual Healthcare Conference - GlobeNewswire

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