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Prediabetes could lead to type 2 diabetes in future - Know its symptoms and causes  |  Photo Credit: iStock Images

New Delhi: Type 2 diabetes has already reached epidemic levels around the world, with millions of people suffering from the condition. While the condition is very common, it is not completely curable, and can only be managed with the help of a healthy diet, regular physical activity and medicines. At the same time, diabetes can increase the risk of other health conditions like heart and kidney problems, if not managed well. In the wake of the COVID-19 pandemic that the world is struggling with right now, people with diabetes have been listed in the 'high-risk' category for risk of infection and complications due to coronavirus.

While many people know about type 2 diabetes, its causes and symptoms, very few are aware of prediabetes a condition that could be the onset of type 2 diabetes. Here is all you need to know about this condition its symptoms, and causes, and how you can keep yourself safe and healthy.

Prediabetes is a condition where your blood sugar or blood glucose levels are high, but not high enough to be termed as type 2 diabetes.

People who develop type 2 diabetes eventually, have prediabetes first. If prediabetes is not checked, it can transpire into type 2 diabetes within about 10 years, experts suggest. People who have prediabetes are at a high risk of developing type 2 diabetes in their life.

Prediabetes is very common, and it does not necessarily mean that one will suffer from type 2 diabetes. Unlike type 2 diabetes, prediabetes is reversible and can be managed with a healthy diet, regular physical activity, and some lifestyle changes.

While there is no particular cause of type 2 diabetes or prediabetes known, certain factors such as family history, genetics, and lifestyle are known to play a role. Obesity, especially around the abdomen, is considered an important factor for the pancreas to not produce insulin, or for the cells in the organs to not use it.

It is important to understand that prediabetes often does not show any signs or symptoms of the condition. Therefore, regular tests, especially for people who re aware that they are at high risk of developing the condition based on the causes listen above, are extremely vital.

Some uncommon symptoms of prediabetes include -

The bottom line is that a healthy diet comprising of high-fibre, nutrient and mineral-rich food, and a healthy lifestyle that comprises of mindfulness and regular exercise is important to stay healthy and keep the risk of diseases at bay, including prediabetes and type 2 diabetes.

Disclaimer: Tips and suggestions mentioned in the article are for general information purposes only and should not be construed as professional medical advice. Always consult your doctor or a professional healthcare provider if you have any specific questions about any medical matter.

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Prediabetes could lead to type 2 diabetes in future - Know its symptoms and causes - Times Now

Coffee can help reduce the risk of developing type 2 diabetes but just filtered coffee, rather than boiled coffee. New research study from Chalmers University of Innovation and Ume University, both in Sweden, show that the option of preparation method influences the health effects of coffee.

Numerous previous studies have actually shown a connection between high coffee intake and a minimized risk of establishing type 2 diabetes. Now, a research study from Chalmers University of Innovation and Ume University, offers new insight into this connection, using an unique approach to help distinguish between the effects of filtered coffee and boiled coffee.

We have identified specific molecules biomarkers in the blood of those taking part in the study, which indicate the intake of different sorts of coffee. These biomarkers are then used for analysis when calculating type 2 diabetes risk. Our results now clearly show that filtered coffee has a positive effect in terms of reducing the risk of developing type 2 diabetes. But boiled coffee does not have this effect, says Rikard Landberg, Professor in Food Science at Chalmers, and Affiliated Professor at the Department of Public Health and Clinical Medicine at Ume University.

With making use of these biomarkers, the researchers had the ability to show that people who drank 2 to 3 cups of filtered coffee a day had a 60% lower threat of establishing type 2 diabetes than people who drank less than one cup of filtered coffee a day. Consumption of boiled coffee had no effect on the diabetes risk in the research study.

Filtered coffee is the most common approach of preparation in many places, including the US and Scandinavia. Boiled coffee in this case refers to an alternative method of coffee preparation often used in Sweden and some other nations, in which coarse ground coffee is simply added straight to boiling water and left to brew for a couple of minutes. All the information used in the research originated from a group of Swedish subjects and was gathered in the early 1990s.

According to Rikard Landberg, many individuals incorrectly think that coffee has only unfavorable impacts on health. This could be since previous research studies have shown that boiled coffee increases the threat of heart and vascular diseases, due to the existence of diterpenes, a type of particle found in boiled coffee.

But it has been shown that when you filter coffee, the diterpenes are captured in the filter. As a result, you get the health benefits of the many other molecules present, such as different phenolic substances. In moderate amounts, caffeine also has positive health effects, he says.

The question is whether diterpenes likewise negatively affect sugar metabolic process and are therefore the cause of why boiled coffee does not help decrease the threat of diabetes, in the way that filter coffee does. The scientists still can not say the specific nature of the link.

Numerous other types of coffee preparation were not specifically examined in the research study, such as instant, espresso, cafetire, and percolator coffee. These kinds of coffee were not typical amongst the Swedish research study population when the data was collected.

However considered that espresso coffee, from traditional espresso machines or the now popular coffee-pods, is also brewed without filters, Rikard Landberg believes the health effects could therefore resemble boiled coffee, in regards to the threat of type 2 diabetes. Coffee made in a cafetire, or French press, is prepared in a comparable method to boiled coffee, so it may also not have the positive impact of reducing type 2 diabetes danger. It is uncertain whether instantaneous coffee, the most popular enter the UK, would be more similar to filtered or boiled coffee in this regard.

However the researchers are careful to note that no conclusions can be drawn yet regarding these other preparation techniques. Rickard Landberg likewise stresses that the health effects of coffee do not depend exclusively on if it is filtered or not. They likewise differ with how the coffee beans, and the drink in general, are handled.

To differentiate the diabetes risk for boiled and filtered coffee, a new strategy called metabolomics was used, in combination with timeless dietary surveys. Metabolomics makes it possible to identify the blood concentration of specific particles from a given food or drink and utilize that as an unbiased measurement of consumption instead of simply depending on self-reported consumptions from the questionnaires, which are prone to big mistakes.

Metabolomics is a fantastic tool, not just for capturing the intake of specific foods and drinks, but also for studying the effects that that intake has on peoples metabolism. We can derive important information on the mechanisms behind how certain foods influence disease risk, says Lin Shi, Postdoctoral researcher and the lead author of the study.

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Filtered Coffee can help slow the risk of developing type 2 diabetes - Drew Reports News

One sign to note is having sweet-smelling pee.

A person with uncontrolled diabetes may have blood glucose levels that are dangerously high, and as the body tries to get rid of the extra glucose in the urine it can cause a sweet smell, according to Medical News Today.

The site adds: People with sweet-smelling urine due to diabetes may notice other symptoms, including exhaustion, extreme thirst, appetite changes and unexplained weight loss.

Its important to note other conditions can cause a persons pee to smell.

READ MORE:How to live longer: Consume this spice to reduce heart disease risk and inhibit cancer

Smelly pee accompanied with lower back pain, pain when peeing and blood in pee can be caused by kidney stones, says the NHS.

And smelly pee accompanied by yellow skin and eyes (jaundice, tummy pain, nausea and vomiting can be a sign of liver failure.

But this isnt the only way a persons pee can be affected.

Polyuria is a condition where the body urinates more than usual and can be triggered by Type 2 diabetes.

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A person may also pass excessive or abnormally large amounts of urine each time they urinate.

Polyuria is defined as the frequent passage of large volumes of urine more than three litres a day compared to the normal daily urine output in adults of about one to two litres, explains Diabetes.co.uk.

It is one of the main symptoms of diabetes (both type 1 and type 2 diabetes) and can lead to severe dehydration, which if left untreated can affect kidney function.

The most common sign of polyuria is producing abnormally large volumes of urine at regular intervals throughout the day and night.

The charity adds: Having some of the signs and symptoms of diabetes doesnt mean you definitely have the condition, but you should always contact your GP, just to make sure.

A healthy diet and keeping active will help you manage your blood sugar level, says the NHS.

It advises you eat a wide range of foods, including fruit, vegetables and some starchy foods like pasta, to keep sugar, fat and salt to a minimum, and to eat breakfast, lunch and dinner every day - do not skip meals.

It also advises you do 2.5 hours of activity a week.

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Type 2 diabetes symptoms: The sign in your urine you could have the chronic condition - Express

Wearing face maskswith reading glasses is now worry-free-thanks to The University of Wisconsin and YouTube's Lennon Rodgers, who posted a do-it-yourself (DIY) hack in his channel that demonstrates the step-by-step procedure for people to create their own 'Badger Seals.' The university's innovation will help prevent hot steam from human breath exiting the mask and fogging-up lenses in the process.

Many people who wear reading and shaded glasses, together with the required face masks, who stroll around the streets and public areas, are bothered by the fog that it causes to the lenses. This fog is bothersome as it requires users to remove and clean the glasses to clear-up vision.

The American Academy of Opthalmology (AAO)recognizes this dilemma for people who wear prescription glasses because this is considered to be a potential danger when walking, driving, and other essential actions are visually impaired due to the lenses' fog.

The University of Wisconsin-Madison College of Engineering releases a reporton the "mask fitters" that prevent people from fogging up their glasses. The university referred to this as the "Badger Seal" and shared the step-by-step procedure on various Open Document sources in Google Slidesand PDFs.

David Rothamer and Scott Sanders spearheaded this design for the public to use and create on their own. The researchers and engineers even provided 3D images available for 3D printing for ease of creation.

ALSO READ: CDC: COVID-19 Vaccines Will Administer on 'Day of the Dead' as Experts Fear the Rush

The Badger Seal is a soft and flexible mask fitter frame that locks the mask in place, significantly improving the cover's seal in a person's face. The currently available face masks are fitted for the general public and do not cater to specific facial shapes.

This leaves some people to have some gaps for air to enter and escape, thus defeating the purpose of protection against viruses, prominently, the Novel coronavirus, or COVID-19.

The University of Wisconsin promises that this design will enhance a 3-layer face mask's protection more effectively. Badger Seals can also be manufactured cheaply and easily by people who have no experience creating various designs.

Here is a rundown of the components needed to create a DIY Badger Seal right at your home's comforts.

Tools:

Materials

The researchers, along with Lennon Rodgers, created instructional videos that demonstrate the Badger Seals' creation and function. There are three types of seals (A, B, and C) to choose from according to your preference and material availability.

The three types of Badger Seals offer the same design of frames but are intended for different face sizes and materials. Type A is designed for the average facial size with the tie-on design, Type B is intended for those with slightly bigger face size (those with beards), and Type C is intended for those who will utilize earloops for easier installation.

ALSO READ: Elon Musk Gets German Minister's Full Support in Trip to Berlin Gigafactory Despite Anti-Tesla Protests

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Written by Isaiah Alonzo

2018 TECHTIMES.com All rights reserved. Do not reproduce without permission.

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WATCH: This Badger Seal Hack Promises a Fog-Free Experience While Wearing Face Masks; Vision Impaired No More - Tech Times

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Global Tissue Ablation Market 2020 by Company, Regions, Type and Application, Forecast to 2025Boston Scientific, Abbott, Johnson & Johnson,...

This report includes the overall and comprehensive study of the Opthalmology Pacs market with all its aspects influencing the growth of the market. This report is exhaustive quantitative analyses of the Opthalmology Pacs industry and provides data for making strategies to increase the market growth and effectiveness. The Global Opthalmology Pacs market 2020 research provides a basic overview of the industry including definitions, classifications, applications and industry chain structure. The Global Opthalmology Pacs market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status.

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HospitalsAmbulatory Surgical Center (ASCS) & Specialty ClinicsOthersBy Type, market is segmented into:

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Opthalmology Pacs Market 2020 Global Innovations, Competitive Analysis, New Business Developments and Top Companies Global Forecast to 2026 -...

A new research study from Data Bridge Market Research with title Microbiome Sequencing Insights 2019, Global Analysis and Forecast to 2026 provides an in-depth assessment of the Microbiome Sequencing including key market trends, upcoming technologies, industry drivers, challenges, regulatory policies, key players company profiles and strategies. Some of the leading key players profiled in this study:BIOLOG; Cosmosid Inc; Illumina, Inc.; Metabiomics Corp.; Oxford Nanopore Technologies; BaseClear B.V.; BGI; BioMathematica; Charles River; CoreBiome, Inc.; Clinical-Microbiomics A/S; Diversigen; Eurofins Scientific; LABCYTE INC.; Leucine Rich Bio Pvt Ltd.; Microbiome Insights; MICROBIOME THERAPEUTICS, LLC; Thermo Fisher Scientific Inc.; Molzym GmbH & Co. KG; Norgen Biotek Corp.; Phase Genomics Inc.; QIAGEN; Resphera Biosciences, LLC; Shoreline Biome; StarSEQ GmbH; Viome, Inc.; Zymo Research among others.

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Global microbiome sequencing marketis expected to rise to an estimated value of USD 3455.33 million by 2026, registering a healthy CAGR in the forecast period of 2019-2026. This rise in market value can be attributed to the focus of various researchers on utilization of this technology in the development of personalized medicine and dietary applications.

Key Developments in the Market:

Market Drivers

Market Restraints

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Global Microbiome Sequencing Market Growth Drivers, Demands, Business Opportunities and Demand Forecast to 2026|Clinical-Microbiomics A/S; Diversigen;...

Adenovirus vectors have become a popular delivery platform for gene therapy and vaccines. However, many people have preexisting immunity against adeno-associated viruses (AAVs), which could significantly reduce vector uptake and therefore render the therapeutic cargo they carry ineffective. Even in individuals that lack preexisting immunity, the first inoculation of a vector could induce immune responses that can thwart repeat administration.

To overcome that obstacle, scientists at the University of Pittsburgh created a CRISPR-based system that they say could effectively prevent immunity to the viral vector. The CRISPR tool briefly suppressedgenes that are related to anti-adenovirus antibody production and helped achieve better gene therapy uptake in mice. The results werepublished in Nature Cell Biology.

Samira Kiani, the studys co-senior author, has co-founded SafeGen Therapeutics with the goal to bring this platform to the clinic to enable safer gene therapy.

Accelerate Clinical Operations Across Sponsors, CROs, and Partners

The most advanced life sciences organizations know that digital innovation and multi-platform integrations are essential for enabling product development. New platforms are providing the life sciences industry with an opportunity to improve the efficiency of clinical trials and reduce costs while remaining compliant and reducing risk.

Many clinical trials fail because of the immune response against AAV gene therapy, Kiani said in a statement. And then you cant re-administer the shot because people have developed immunity.

One recent case in point is CanSino Biologics adenovirus type 5 (Ad5)-vectored COVID-19 vaccine. In the shots phase 2 trial, about half of the participants had high levels of preexisting Ad5 neutralizing antibody. These individuals were found to produce significantly lower immune responses to the vaccine than did those with low preexisting anti-Ad5 immunity.

Kiani and colleagues set out to modify the gene expression that's associated with an immune response against AAV, but only temporarilyjust long enough for the therapeutic content to do its jobbecause they didnt want to tamper with normal immune functions.

RELATED:Hopkins team invents non-viral system for getting gene therapy into cells

CRISPR, which can edit out a defective gene or add in a functional one, is already being explored as a gene therapy strategy. So the University of Pittsburgh researchers figured they could hit two birds with one stone by utilizing CRISPR to also control the immune response.

They treated mice with the CRISPR-based system and then re-exposed them to AAV, and the animals didnt make more antibodies against the virus. The treated mice were more receptive to subsequent AAV-delivered gene therapy than were the control animals, the team reported.

Gene therapies hold promise as potential cures to many hard-to-treat diseases that would otherwise require chronic treatment. So scientists have been trying to perfect their transport vehicles for better safety and efficacy.

A team led by scientists at the San Raffaele Telethon Institute for Gene Therapy in Italy, for example,attached the CD47 protein to the surface of lentiviral vectors so they could escape detection and destruction by the immune system. And researchersfrom Johns Hopkins University developed a nonviral delivery system that uses a polymer nano-container, whichreleases the therapy once inside the cell.

Now, Kianihopes to validate the CRISPR-based technology at SafeGen. In addition to its potential as a gene therapy delivery platform, the researchers also found that their method can prevent or treat sepsis in mice, suggesting it might also be useful for treating inflammatory conditions.

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Using CRISPR to improve viral vectors for gene therapy - FierceBiotech

Gene therapy generally relies on viruses, such as adeno-associated virus (AAV), to deliver genes into a cell. In case of CRISPR-based gene therapies, molecular scissors can then snip out a defective gene, add in a missing sequence or enact a temporary change in its expression, but the bodys immune response to AAV can thwart the whole endeavor.

To overcome that obstacle, researchers at the University of Pittsburgh created a system that uses CRISPR in a different way. Their system briefly suppresses genes that are related to AAV antibody production so the virus can deliver its cargo unimpeded. These results published today inNature Cell Biology.Many clinical trials fail because of the immune response against AAV gene therapy, said study co-senior author Samira Kiani, associate professor of pathology in Pitt'sSchool of Medicineand member of thePittsburgh Liver Research Center(PLRC) andMcGowan Institute for Regenerative Medicine. And then you cant readminister the shot because people have developed immunity.

So Kiani and her long-time collaborator Mo Ebrahimkhani, associate professor of pathology at Pitt, member of PLRC and the McGowan Institute, set out to modify gene expression related to the bodys immune response to AAV. But this gene is important for normal immune function, so the researchers didnt want to shut it down forever, just tamp it down momentarily.

Since CRISPR is such a convenient system for editing the genome, the pair figured they would put it to use for altering the master switches that orchestrate genes involved in immune response.

Were hitting two birds with one stone, said Ebrahimkhani. You can use CRISPR to do your gene therapy, and you can also use CRISPR to control the immune response.

When the researchers treated mice with their CRISPR-controlled immune suppression system and then exposed them to AAV again, the animals didnt make more antibodies against the virus. These animals were more receptive to subsequent AAV-delivered gene therapy compared to controls.

Beyond gene therapy, the study also shows that CRISPR-based immune suppression can prevent or treat sepsis in mice, highlighting the potential for this tool to be broadly useful for a range of inflammatory conditions, including cytokine storm andacute respiratory distress syndrome, both of which can crop up with COVID-19, though more studies are needed to engineer safety features.

The main goal of this study was to develop CRISPR-based tools for inflammatory conditions, said study lead author Farzaneh Moghadam, a PhDstudent in Kianis lab. But when we looked at bone marrow samples, we saw that the group treated with our tool showed a lower immune response to AAV compared to the control group. That was very interesting, so we started exploring how this tool contributes to antibody formation against AAV and could potentially address safety and efficacy concerns with gene therapy trials.

Kiani co-founded SafeGen Therapeutics with the goal of bringing this technology to the clinic.

This study was supported by National Institute of Biomedical Imaging and Bioengineering, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, and a DARPA Young Faculty Award.

Reference: Moghadam F, LeGraw R, Velazquez JJ, et al. Synthetic immunomodulation with a CRISPR super-repressor in vivo. Nature Cell Biology. 2020;22(9):1143-1154. doi:10.1038/s41556-020-0563-3.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Continued here:
Editing the Immune Response To Aid the Delivery of Gene Therapy Cargo - Technology Networks

TipRanks

As fears of a tech bubble and stretched valuations become the talk of the town, investors are turning to Wall Street titans for guidance, namely Ken Griffin. Founding hedge fund Citadel in 1990, the firm now boasts over $35 billion worth of assets under management.As a 19-year-old sophomore at Harvard University, Griffin began trading from his dorm room with a fax machine, computer and phone. Now, the CEO of Citadel, whose net worth stands at $15.5 billion, is known as one of the Wall Street greats. Looking at the funds performance during the COVID crisis, its even more clear why Griffin has legendary status.Unlike the average hedge fund, which had a negative return of between 3-4% in the first half of 2020, Citadels flagship Wellington fund saw its returns land between 13-14% for the same period.Bearing this in mind, we wanted to take a closer look at three stocks Citadel snapped up recently. Using TipRanks database, we found out that each ticker has earned a Strong Buy consensus rating from the analyst community. Not to mention all three of them boast massive upside potential.AVEO Pharmaceuticals (AVEO)Hoping to provide better outcomes for patients, AVEO Pharmaceuticals advances targeted medicines for oncology and other unmet medical needs. Following an important regulatory milestone, its no wonder all eyes are on this healthcare name.Griffin is among those singing AVEOs praises. Increasing its holding by a whopping 2,357%, Citadel bought up 383,720 shares in Q2. With the total position now landing at 400,003 shares, it is valued at $1,824,013.H.C. Wainwright analyst Swayampakula Ramakanth reminds investors that on June 1, the FDA accepted the NDA for tivozanib, the company's lead candidate, for review, based on the fact that the TIVO-3 study reported positive final overall survival (OS) data. In the study, AVEOs therapy was compared to sorafenib, marketed as Nexavar by Bayer, for the treatment of advanced renal cell carcinoma (RCC) in the third and fourth-line settings.Looking more closely at the data, which was presented at the ASCO 2020 virtual meeting, the final OS analysis resulted in an overall hazard ratio (HR) of 0.97, which favored tivozanib. Ramakanth was encouraged by the OS results as they suggest tivozanib at least has a similar overall relative risk of deaths compared to sorafenib.Considering that TIVO-3 study met both the primary endpoint of progression free survival (PFS) and the secondary endpoint of overall response rate (ORR), with comparable OS to the active comparator, we believe tivozanib would likely get a green light for the U.S. approval, which could be a major catalyst in the next 12 months, Ramakanth opined.Adding to the good news, the dose escalation for the Phase 1b/2 DEDUCTIVE study, evaluating tivozanib in combination with durvalumab, a monoclonal antibody against PD-L1 marketed as Imfinzi by AstraZeneca in hepatocellular carcinoma (HCC), has been wrapped up, with it progressing to Phase 2. As the CDC estimates about 33,000 patients suffer from liver cancer every year in the U.S., Ramakanth sees an additional opportunity.To this end, Ramakanth rates AVEO a Buy rating along with a $12 price target. Should his thesis play out, a potential twelve-month gain of 163% could be in the cards. (To watch Ramakanths track record, click here)Other analysts dont beg to differ. 3 Buy ratings and no Holds or Sells have been assigned in the last three months. So, the word on the Street is that AVEO is a Strong Buy. The $15 average price target is more aggressive than Ramakanths and implies 229% upside potential. (See AVEO stock analysis on TipRanks)IDEAYA Biosciences (IDYA)Next up we have IDEAYA Biosciences, an oncology-focused precision medicine company that develops targeted therapeutics by using molecular diagnostics. Based on the strength of its technology, this name has scored several fans.Reflecting a new position for Griffin's Citadel, the fund pulled the trigger on 248,005 shares in Q2. As for the value of this holding, it comes in at $2,881,818. Writing for Northland Capital, analyst Tim Chiang believes shares are undervalued based on the future potential of its precision medicine oncology pipeline, which targets specific biomarkers. Expounding on this, he stated, IDEAYA is applying its capabilities across multiple classes of precision medicine, including direct targeting of oncogenic pathways and synthetic lethality which represents an emerging class of precision medicine targets.Part of what makes IDYA a stand-out, in Chiangs opinion, is the fact that its preclinical programs use its synthetic lethality (SL) platform, which targets tumors with MTAP gene deletion and homologous recombination deficiency (HRD) including BRCA mutations.We believe the longer-term upside potential with IDYA shares is significant given the potential utility of SL. The first clinically validated SL gene pair was PARP-BRCA1/2, and based on the efficacy of PARP inhibitors, the SL approach to treating cancer has achieved substantial commercial validation, the analyst explained.To back this up, Chiang points out that several PARP inhibitors have already been approved for the treatment of tumors with BRCA and other DNA damage repair alterations, including ovarian, breast and pancreatic cancers. These inhibitors include AstraZeneca's olaparib, GlaxoSmithKline's niraparib, Pfizer's talazoparib and Clovis rucaparib. He added, We estimate these four drugs generated over $1.6 billion in worldwide sales in 2019 and are expected to reach over $6 billion in sales by 2024.It should be noted that multiple IND filings are set to come within the next 4-12 months, with IDYAs lead SL candidate, IDE397, which was designed to inhibit MTAP and MAT2A and thus cause the death of cancerous tumor cells, entering the clinic in 2021.It should come as no surprise, then, that Chiang joined the bulls. To start off his IDYA coverage, he puts an Outperform rating and $28 price target on the stock. This target implies a possible twelve-month rise of 141% could be on the horizon. (To watch Chiangs track record, click here)Similarly, the rest of the Street is getting onboard. 5 Buy ratings assigned in the last three months add up to a Strong Buy analyst consensus. In addition, the $25.20 average price target puts the potential twelve-month gain at 116%. (See IDYA stock analysis on TipRanks)Ocular Therapeutix (OCUL)Using its patented bioresorbable hydrogel-based formulation technology, Ocular Therapeutix develops cutting-edge therapies for diseases and conditions of the eye. The progress of its clinical programs has caught Wall Streets attention, with some arguing that now is the time to get in on the action.Griffin and Citadel didnt want to miss out on an opportunity. Snapping up 161,032 shares during Q2, the hedge fund gave the holding a 272% boost. The total position is now comprised of 220,269 shares and is valued at $1,718,098.Representing Raymond James, 5-star analyst Dane Leone cites the potential to address the unmet needs in the dry eye disease indication as a key component of his bullish thesis. The company boasts two assets targeting the condition, OTX-CSI (chronic) and OTX-DED (acute). OTX-CSI incorporates the FDA-approved immunomodulator cyclosporine as the active drug in the intracanalicular insert, which is released for an estimated three months to increase tear production.When it comes to OTX-DED, Leone argues OCUL was clever in pursuing the development of OTX-DED (a low dose form of DEXTENZA, a corticosteroid intracanalicular insert placed in the punctum, a natural opening in the eye lid, and into the canaliculus to deliver dexamethasone to the ocular surface for up to 30 days without preservatives) for the treatment of episodic dry eye, as the prior safety data from the DEXTENZA approval enabled management to file a Phase 2-enabling IND by YE20.Speaking to the possible opportunity here, both products could provide revenue generation for physicians in the treatment of dry eyes using procedure CPT code 0356T, which could provide incentive for rapid adoption in the dry eye space that is currently valued at $5.1 billion, in Leones opinion.As for the clinical and regulatory pathway, the Phase 2 trial for OTX-DED is set to kick off in 2H21, slightly after OTX-CSI, which will see a Phase 2 clinical trial evaluating two different formulations initiated by 4Q20. Based on the short treatment duration of OTX-DED, management thinks it will still be brought to market first.To this end, Leone remains optimistic about the companys long-term growth narrative. As a result, he rates OCUL a Strong Buy along with a $15 price target. This figure implies shares could rise 89% in the year ahead. (To watch Leones track record, click here) Turning now to the rest of the Street, other analysts are on the same page. With 100% Street support, or 3 Buy ratings to be exact, the consensus is unanimous: OCUL is a Strong Buy. The $13.50 average price target brings the upside potential to 70%. (See OCUL stock analysis on TipRanks)To find good ideas for stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Gene Therapy Market is Forecast to Reach US$ 6.42 billion in 2024, Witnessing Growth at a CAGR of 19.29% Over 2020-2024 - Yahoo Finance

Gyros Protein Technologies AB has announced the introduction of Gyrolab AAVX Titer Kit for adeno-associated virus (AAV) vector titer determination in AAV vector manufacturing. The new Gyrolab AAVX Titer Kit for physical titer determination, one of the critical quality attributes (CQAs) of the FDA process validation guidelines, has a broad applicability across numerous AAV serotypes and adds to the Companys wide range of ready-to-use kits that are already used by scientists and bioengineers in the rapidly growing cell and gene therapy market, where AAV vectors are commonly used.

Gyrolab AAVX Titer Kit supports scientists in the development and production of cell and gene therapies to maximize performance and productivity, and reduces the time needed to produce patient treatments. With compressed development timelines for these therapies, the new kit generates results 4 times faster and requires 10 times less sample compared to traditional ELISA methods, accelerating AAV vector workflows and enabling high quality data to be produced from small sample volumes. Featuring an analytical range of >3 logs, Gyrolab AAVX Titer Kit also minimizes repeat testing compared to ELISA.

Gyrolab AAVX Titer Kit is based on a highly selective AAVX affinity ligand developed with the Thermo Scientific CaptureSelect technology by Thermo Fisher Scientific. These ligands are also the basis of POROS CaptureSelect AAVX Affinity Resin, which is frequently used to purify AAV viral vectors. The AAVX ligand binds with high affinity and selectivity to native and recombinant AAV particles of various serotypes (AAV1 to AAV8, AAVrh10).

Read more:
Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks

The development prediction report titled Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 by Company, Regions, Type and Application, Forecast to 2025 focuses on thoughtful insights and facts relating to the market. The report investigates the most recent market patterns such as market development openings, size, share, and drivers. The report throws light on the markets historical data, key vendors, region-wise market, and projections for 2020 to 2025 time-period. Report authors have categorized global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market segments, regions, and product types and distribution channels to provide market analysis and information. All the relevant points of interest market product type, producing price, scope, applications are covered in the report. In the later section, market dynamics are covered including market growth factors, limitations, market opportunities, and challenges are mentioned.

NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

Competitive Rivalry:

The report offers a comprehensive study of prime players operating in this global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market by highlighting their product description, business outline, and business strategy. The report conjointly encompasses the amount of production, future demand, and also the health of the organization. Later, the report highlights all the recent developments, product launches, joint ventures, merges, and accusations by the top players. The leading players are also covered with product description, business outline, and production, company profile, product portfolio, product/service price, capacity, sales, and cost.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/83503

The report analyses every sub-segment regarding the individual growth trends, contribution to the total market, and the upcoming forecasts. For the forecast period, the report offers estimates of global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market value and volume. The report discusses key drivers, market challenges, cost structure, barriers, technology, regions, and applications. It spotlights on effective manufacturing methods, production volume, manufacturing facilities, capacities, pricing analysis, product description.

Top key players involved in the industry are: BioReliance, Richter-Helm, UniQure, Cobra Biologics, MassBiologics, Oxford BioMedica, Lonza, MolMed, FinVector, FUJIFILM Diosynth Biotechnologies, Brammer Bio, bluebird bio, Aldevron, Spark Therapeutics, VGXI, Biovian, Eurogentec, Novasep, PlasmidFactory, Cell and Gene Therapy Catapult, Vigene Biosciences

Segmentation by type and analysis of the market: AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors

Segmentation by application and analysis of the market: Cancers, Inherited Disorders, Viral Infections, Others

The report offers an exhaustive geographical analysis of the global market, covering important regions such as: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

Moreover, the report analyzes the worlds main regions contributing to the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market development. These regions are assessed the product price, profit, capacity, production, supply, demand, and market growth rate and forecast, etc. Additionally, manufacturing processes, cost structures, import/export consumption, supply and demand figures, cost, price, revenue, and gross margins are analyzed.

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Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 Analysis, Types, Applications, Forecast and COVID-19 Impact...

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

Originally posted here:
Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - Scientect

LONDON Gyroscope Therapeutics Ltd. is poised to move the field of ocular gene therapy on from the treatment of inherited rare diseases to address more common eye conditions, after receiving FDA 510(k) clearance for its Orbit SDS subretinal delivery device.

Orbit SDS uses a cannula to deliver a therapeutic payload to the subretinal space, which it accesses via a suprachoroidal approach route. A microneedle inside the cannula enables targeted dose delivery.

That eliminates the need for a vitrectomy, and is both faster, easier and safer than the surgical procedure, removing concerns about gene therapy vectors leaking from the subretinal space.

Although vitrectomy has been used to deliver several gene therapies, it is not practical for treating hundreds of thousands of patients with dry age-related macular degeneration (AMD) and other common eye diseases. FDA 510(k) clearance of Orbit SDS now opens the way to scale up administration of ocular gene therapies to much larger indications.

The FDA clearance creates the potential to speed up development and widen patient access. In addition to a replacing a defective gene with a functional one, it will be possible to expand into the routine delivery of therapeutic proteins.

The company says it will use the device in its own clinical trials and make it available to other companies developing cell and gene therapies for eye diseases.

Charlotte Arnold, vie president of corporate affairs at Stevenage, U.K.-based Gyroscope told BioWorld the company now plans to integrate Orbit SDS into its GT005 program in dry AMD. We anticipate starting to generate data delivering GT005 using the Orbit SDS in a planned future cohort in the ongoing phase I/II Focus trial, she said.

Unlike the wet form of AMD that can be treated with anti-VEGF antibodies to prevent neovascularization, there is no therapy approved for dry AMD, which is both a cause of significant visual impairment and a precursor to wet AMD.

GT005 uses an adeno-associated virus to deliver the gene encoding an endogenous anti-inflammatory factor, to restore the balance of a complement system known to be hyperactivated in AMD. The aim is that a one-off treatment will prevent further degeneration.

The FDA nod is an important component of developing gene therapies to help preserve sight, said Khurem Farooq, CEO of Gyroscope. The Orbit [device] is exquisitely designed to target the subretinal space, with the aim of providing precise and consistent dosing, Farooq said.

The clearance for Orbit SDS rests on a phase IIb study of palucorcel (CNTO-2476), in the treatment of geographic atrophy of age-related AMD, an advanced stage of AMD, characterized by progressive loss of retinal pigment epithelium. That leads to loss of photoreceptors, resulting in visual disturbance and difficulty adjusting to low light conditions. Central vision is lost progressively.

The Orbit device was used in the phase I/II trial after initial studies in which palucorel was administered by retinotomy showed there was a risk of retinal detachment. A further study used subretinal delivery, but the technique was judged too technically difficult to take forward.

That led on to the development of Orbit SDS, which was purpose designed for subretinal delivery.

In the 21-patient phase I/II palucorel study the investigators reported that administration to the subretinal site was achieved successfully in most patients and [was] not associated with any moderate or severe, or serious ocular adverse events. The investigators were unable to deliver cells in 3 of 21 participants. Most adverse events were mild and resolved within one month.

Vitrectomy can be associated with an increase in intraocular pressure and accelerated formation of cataracts. No such effects were seen in the Orbit SDS study.

Palucorel is a cell therapy derived from human umbilical tissue that expresses anti-inflammatory factors including interleukin-6, brain-derived growth factor, fibroblast growth factors, and anti-angiogenic thrombospondins. While it showed positive effects in two earlier trials, there were no significant improvements in vision in the Orbit SDS-administered trial.

However, the investigators say, This first in human trial of a suprachoroidal approach for delivery of subretinal liquids was successful from a surgical perspective and clearly demonstrates that subretinal delivery via the suprachoroidal space is feasible.

Gyroscope acquired the Orbit SDA device when it merged with its developer, Orbit Biomedical of Philadelphia in April 2019, to become a fully integrated retinal gene therapy company, with clinical, manufacturing and delivery capabilities.

Orbit Biomedical, now renamed Gyroscope USA Inc., is an ISO 13485 medical device company certified to design, manufacture and distribute ophthalmic surgical instruments.

Following on from the FDA clearance, Arnold said Gyroscope is pursuing a CE mark in Europe for Orbit SDS as well. We also plan to enter into licensing and collaboration arrangements for use of the Orbit SDS by other companies for delivery of gene and cell therapies being developed to treat eye diseases, she said.

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Gyroscope Therapeutics FDA clearance puts subretinal delivery device in Orbit - BioWorld Online

Non-Hodgkin lymphoma (NHL) is among the most common cancers in the United States. In 2020, an estimated 77,240 Americans will be diagnosed with the disease and about 20,000 people will die from it.1 Seventy-two percent of patients live for at least 5 years after diagnosis.2 However, the success of treatment varies widely across the many subtypes of NHL. Patients diagnosed with diffuse large B-cell lymphoma (DLBCL), the most common subtype, have a 5-year survival rate of 63% for all disease stages combined. By contrast, follicular lymphoma (FL) has a 5-year survival rate of 88% for all stages combined. Patients with mantle cell lymphoma, one of the more difficult-to-treat subtypes, have an average survival time of less than 5 years.3

But the treatment landscape is transforming across the entire spectrum of NHL. Novel immunotherapies and small molecule inhibitors are offering both previously treated and untreated patients entirely new options and new combinations. At the American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program, researchers led by Jeremy Abramson, of Massachusetts General Hospital in Boston, provided a compelling overview of these new and upcoming treatments.4 Their overview, published in the ASCO Educational Book, offered a detailed and vital look at the present and future of NHL treatment.

CAR T-cell immunotherapy has been hailed as a major game-changer for some blood cancers.5 For aggressive B-cell lymphomas, such as DLBCL, anti-CD19 chimeric antigen receptor T cells (CAR-T) are a new option for patients who have relapsed following chemoimmunotherapy or autologous stem cell transplant (ASCT). For this patient group, which has a median overall survival of about 4 months, anti-CD19 CAR-T agents have elicited durable remissions in about 40% of patients.5-7 Clinical trial data has led to the approval of axicabtagene, ciloleucel, and tisagenlecleucel by both the US Food and Drug Administration and the European Medicine Agency. CAR T-cells offer curative intent therapy to patients with relapsed DLBCL who are not eligible for stem cell transplant, Dr Abramson told Cancer Therapy Advisor. Previously these patients only had palliative options available.

Some clinicians envision CAR-T as a potential first-line therapy for patients with DLBCL who relapsed after first-line chemoimmunotherapy. These patients, noted Anton Hagenbeek, MD, PhD, professor of Hematology at Amsterdam University Medical Centers, who was not involved with the review paper, represent one of the highest unmet needs in the treatment of lymphoma to date. And Helen Heslop, MD, who directs the Center for Cell and Gene Therapy at Baylor College of Medicine in Houston, Texas, noted that trials for first-line CAR-T therapy are already underway in acute lymphoblastic leukemia. Not everyone sees this potential. First-line treatment in NHL is generally very effective and much less expensive than CAR T-cells, explained Edward Copelan, MD, who chairs the Department of Hematologic Oncology and Blood Disorders at Carolinas Healthcare System in Charlotte, North Carolina, and who was not an author of the review. Though Dr Copelan emphasized that patients at extremely high risk of relapse following standard immunochemotherapy may respond well to CAR-T therapy.

Dr Abramson and co-authors highlighted a difficult conundrum with regard to DLBCL. Although the addition of rituximab to cyclophosphamide, doxorubicin, hydrochloride, and vincristine sulfate (CHOP) chemotherapy is curative for most patients, the number of patients who may be cured after relapse has declined. Thus improvements in second-line therapy are desperately needed. Several clinical trials are currently evaluating anti CD19 CAR-T for primary refractory or early relapsed aggressive B-cell lymphoma compared with traditional salvage therapy or ASCT. The same approach is being studied for relapsed DLBCL patients who are not transplant candidates.

Whether CAR-T therapy will find a place on the first line of care is another current question. The cost and logistics of CAR-T therapy make it unlikely that this approach will replace R-CHOP as the standard initial treatment, Dr Abramson and colleagues noted. I do not think CAR T-cells will replace frontline chemotherapy, Dr Abramson said. However, the authors note that for patients who do not respond well to initial treatment, such an approach may make sense a possibility that is now being examined in the clinical trial setting. Dr Hagenbeek is more certain about the first-line role for CAR-T therapy, based on the dismal prognosis of DLBCL that is refractory to R-CHOP.

On the subject of CAR-T therapy as first-line therapy, the authors pay special attention to so-called double-hit lymphomas (DHLs), also known as double expressor lymphomas. According to a multicenter study published in 2017, R-CHOP is curative for more patients than conventional wisdom has held.9 Clinical trials are currently investigating the use of anti-CD19 CAR-T early for patients who consistently test positive for disease on PET scans during initial therapy, though Dr Abramson and colleagues call for caution with this approach because the prognostic value of PET/CT scans is controversial. Dr Hagenbeek is optimistic about the potential for CAR-T therapy to improve the prognosis for patients with double- and even triple-hit lymphomas, if applied in the first complete, PET-negative metabolic remission. Because these patients have a relatively small tumor load, this approach could, said Dr. Hagenbeek, completely eradicate minimal residual disease. Its the double-hit patients who Dr Copelan sees as the likeliest candidates for first-line CAR T-cell treatment.

Read more here:
Targeted Therapy for Non-Hodgkin Lymphoma: Current Progress and Future Plans - Cancer Therapy Advisor

Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression

Translation of metabolic correction to clinical outcomes in first two patients continues to support potential of hematopoietic stem cell gene therapy in a second neurometabolic disorder

Data support planned initiation of registrational trial in 2021

BOSTON and LONDON, Sept. 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at theSan Raffaele Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today's results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up, saidMaria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. "These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigationalex vivoautologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard

Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Ourex vivoautologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy inMilan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be successfully developed, approved or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates or that long-term adverse safety findings may be discovered; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedJune 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations +1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - GlobeNewswire

The end of the day often comes too fast, and many of us may find ourselves not quite ready to settle down. There may be those last-minute emails to send, dishes to do, others in the household who need your attention, or anxieties about tomorrows to-do list. All of that buzz makes it tough to drift off to sleep.

Thats where before-bed yoga comes in. The gentle physical movement is relaxing itself, and the principles yoga is founded on gratitude, self-compassion, and contentment when practiced before bed can be calming, too, helping to promote sleep, says Carol Krucoff, a yoga instructor certified by the International Association of Yoga Therapists and Yoga Alliance and a yoga therapist at Duke Integrative Medicine in Durham, North Carolina.

RELATED: 9 Ways Practicing Yoga Benefits Your Health and Well-Being

Restorative yoga is becoming more popular, particularly since COVID-19 has people on edge, says Krucoff, who has a gentle yoga class on YouTube. A review and meta-analysis published in May 2020 in the journal BMC Psychiatry looked at 19 studies with nearly 2,000 women with sleep problems and found that, overall, a yoga practice (at any time of the day) improved sleep quality compared with not practicing yoga.

RELATED: The Basics on Hatha, Kundalini, and Other Types of Yoga

Before you try these poses, set the stage for sleep by centering your mind. Krucoff recommends practicing the Three Good Things exercise. The intention is to think about three good things that happened to you that day. It can be as little as having a really great cup of coffee that morning, she says. (For more details, find this how-to from the Greater Good Science Center at the University of California in Berkeley.) It helps alleviate worry and get you into a positive mindset that helps you relax.

Then try these five gentle poses and exercises to get you all set for sleep:

If you can only do one thing to prepare yourself for sleep, take a few minutes to work on your breath, says Krucoff. What shes referring to is the relaxed abdominal breath, also known as the yoga belly breath. During the day, you are likely in the habit of breathing shallowly from your chest, but deeper breaths fill the lungs completely. This triggers a cascade of physiological changes. Your heart rate slows, your blood pressure decreases, and muscle tension eases, she explains.

How to Do It Lying down, put one hand below your belly button. Breathe in through your nose to fill your lungs (your belly should rise). Breathe out through your nose. Repeat for a few minutes.

RELATED: 7 Quick Stretches for Stress Relief You Can Do Right Now

Here, youll take your joints through their full range of motion. This is something thats quieting, releases tension and tightness, and is convenient because it can even be done lying down in bed, says Krucoff. Whats more, it helps bring attention to body sensations, making you consider not just whatever happened with your family or at work, or what was on the news over the course of the day, but also how your body actually feels. Are certain muscles sore? Are certain muscles tired? Many of us spend much of our day in our heads, Krucoff says. This practice guides you back into your body, which is a good way to prep for sleep.

How to Do ItLie down on the floor or in your bed. Circle your ankles. Extend your legs out long then bend your knees. Lift and lower your hips to make circular movements. Bend your elbows, then extend your arms long at your sides. Shrug and circle your shoulders. Repeat as needed and as feels good.

If you have back issues, the knee hug will feel particularly nourishing, Krucoff says. In fact, low back pain is one of the most common reasons for seeing a doctor; it also keeps people out of work, according to the National Institute of Neurological Disorders and Stroke. Working hunched over at a desk all day can cause the ache. This pose will relieve it.

How to Do ItLie down and hug one or both knees into your chest. (Whether or not you do one or both depends on your physical abilities.) If you can hug both knees into your chest at the same time, rock from side to side to massage your spine.

RELATED: The Best Home Remedies for Back Pain

Its common to hold tension in your neck and shoulders, says Krucoff. Its even more common for tightness and pain to creep up here if you spend your day working at computer or staring at your smartphone.

How to Do ItSit on your bed, sitting up straight with good posture. Inhale and bring your shoulders up to your ears and squeeze your arm and shoulder muscles tightly. Exhale and release your shoulders, pulling your shoulder blades downward. Repeat a few times.

If you practice yoga, you know this as Savasana, which is the final pose of class. It looks extremely easy to lie down and do nothing, but its among the hardest poses to master because it requires you to release all physical and emotional tension and let go of mental thought, explains Krucoff. But no need to stress about getting Savasana right: Just lie down, be still, and try to not think about anything in particular and it will help you relax. Krucoff calls it relaxed alertness which might sound contradictory but is really about noticing whatever thoughts and feelings do come up without dwelling on any one particular one.

How to Do ItLie down with your arms at your sides, palms up and relaxed. Close your eyes and focus on the rise and fall of your breath. If you have trouble with intrusive thoughts, acknowledge their presence and picture them floating away.

RELATED: Yoga Poses for Beginners

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Yoga Poses and Exercises to Help You Sleep - Everyday Health

This article was originally published here

Eur J Cancer Care (Engl). 2020 Sep 3:e13299. doi: 10.1111/ecc.13299. Online ahead of print.

ABSTRACT

OBJECTIVE: Over recent decades, supportive care and patient quality of life, advocated by dedicated guidelines, have become a core focus of the concept of integrative medicine. The Calista 2 survey was conducted in France between September 2016 and October 2017 among oncologists and their patients being treated for early breast cancer, adjuvant colorectal cancer or advanced lung cancer. The present analysis sought to ascertain, understand and rank the expectations of cancer patients with regard to supportive care.

METHODS: Data were collected from 467 questionnaires from patients recruited by 82 oncologists. Inclusion criteria were patients already on treatment for breast cancer, colorectal cancer or lung cancer. Most supportive care facilities were available at the point of care.

RESULTS: Physicians were mainly seen to offer management of adverse events (81%), and pain (72%), psychological support (56%), and advice on diet/nutrition (49%). Patient uptake of supportive care related essentially to management of adverse events (72%) and pain (61%), diet/nutrition (34%), and self-image improvement techniques (31%). The main unmet needs voiced by patients were information on complementary medicines (28%), management of fatigue (27%), and relaxation techniques (24%).

CONCLUSION: Supportive care was essentially seen to satisfy patient requirements with regard to the management of adverse events and pain. However, patients highlighted the need for a wider access to fatigue management and information on complementary medicine and relaxation techniques.

PMID:32885521 | DOI:10.1111/ecc.13299

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Patient perspectives on supportive care in cancer: Results of the Calista 2 study - DocWire News

Taylor Brune @heartsinbloomhealth is many things. To start, perhaps above all else, she is passionate about healthcare. Brune, who recently survived COVID-19, suffers from chronic autoimmune deficiencies, which began from a bite from a tick. As a result, she has had to learn and research much in the medical world so that she is as well equipped as possible to survive her severe afflictions.

On top of all that, Brune is also a Medical Assistant. In this capacity, she works with healthcare providers as a liaison to patients, in administrative capacities and other roles to ensure the facility operates smoothly. Brune, never one to shy away from a challenge, is also a student continuing her education. She is using her experience as a Medical Assistant to help transition to, one day, becoming a doctor. That is her ultimate aim.

We caught up with Brune to ask her about this long professional journey, her fight with COVID-19, her passion for healthcare and much more.

@heartsinbloodhealth

When and why did you decide you wanted to be a Medical Assistant?

The last ten years, I have dealt with my own health issues and my own health journey of developing chronic disease and autoimmune disease after a bite from a tic. So, when I lost my health, I was engulfed in the medical world and I was a patient 24-7 and having to do research for myself and be my own health advocate. In the process of learning how to heal myself, I grew the passion of wanting to help heal others.

While I was going through treatments, I was like, this is my calling. This is where Im supposed to be. This is why Im having my health issues and going through this huge life transition and transformation. When that realization happened, I decided to learn about medicine and how to switch my degree over to pre-med and integrative health.

Going into medical assisting school, was the first step in my path. And Im going to be a doctor one day no matter how long it takes! No matter what challenges I face, I know that everything Im going through in my own health is helping me transform into the best person that I can be so that I can be the best doctor for patients. Since Ive had the perspective of being a patient for so many years, I know exactly what theyre thinking and feeling.

If youre interested in becoming a medical assistant here is an awesome guide to start your research - it answers questions like,

After you read this interview go to the full Medical Assistant Career Guide.

Show Me Medical Assistant Programs

What was the process like for you to become a Medical Assistant?

First, I prayed a lot about it. I knew I wanted to switch my degree to pre-med but, I also wanted to work in the medical field a lot sooner. Becoming a doctor takes years in pre-med and medical school.

So, I figured the first step to completely immerse myself in the medical field as soon as possible was to complete a medical assistant program and to actually start working in the field that I love so much. Once I graduated from my medical assistant and phlebotomy program I immediately started working in the field. The experience Ive gained has just confirmed that this is where Im supposed to be and I love it!

How long did the process take, what type of schooling did you?

For medical assisting in California , I needed to go to a medical assisting school. I went to a trade school and enrolled into a medical assisting program. Medical assisting programs are more about gaining hands-on experience in an actual doctors office. This is how the program was set up,

The program style was really beneficial to help me to get on-the-job training and also land a great position right after graduation,

At the same time, though, Ive been going to Arizona State University Online to finish my Bachelors degree, which Ill be finishing in the fall. This fall, Ill have my Bachelors as well as my medical assisting diploma.

As a medical assistant its important to be certified. I took a national certification exam and every two years, I complete the required credits and retake the test to keep my certification up to date.

@heartsinbloodhealth

How did you land your first job as a Medical Assistant?

It was actually pretty easy for me because I seem to interview really well with medical places. My first job was working at a naturopathic office. Next, I went to primary care and oncology. So, thats where Ive been working the last year. Now, Im at Scripps Health Hospital in dermatology.

Theres a lot to know for the job and you have a lot of responsibilities - from first-aid to computer work to patient liaison. Do you like having all these aspects to your workday?

I love it! I have gained so much experience including,

Drawing blood is my absolute favorite because I really enjoy direct patient care. But, overall working with different modalities, systems and technologies has taught me so much about the medical field. Each private practice is completely different and the providers are unique in that they offer different specialties and treatments. Its been fun learning all these different skills.

@heartsinbloodhealth

Your long-term goal is to be a doctor. How did you choose becoming a medical assistant for that aim and how has it helped?

There are a few reasons why I chose medical assisting as my first step towards my goal of becoming a doctor,

What do you like least about being a Medical Assistant or the healthcare field, in general?

For my own personal experience, I have an immune compromised self. I just dont like that I pick up illnesses so easily. I even picked up COVID. Flu season is also difficult for me. Im thankful for the fact that everyone is now taking more precaution in the medical field. Ive observed that people are more mindful of sanitizing and patients are wearing masks. It gives me hope that at least this year I will have stronger defenses and not catch as many illnesses. But theres many great things about working as a medical assistant, too. Each person has their own experience.

As a chronic illness patient myself, the one negative that I dont like is that I feel some doctors dont have time to really hear their patients. When Im a doctor, that is something that I really want to change. However, Ive been really fortunate to work with providers who are integrative in their mindset and do give their patients time. Even now, working with providers who arent integrative, they still give patients time and hear them. Ive been really fortunate to work with good providers. But, from the perspective of a patient, I had to go through multiple providers to get to good ones - and, I didnt like that.

@heartsinbloodhealth

What advice do you have for other people looking to become a Medical Assistant today - first steps, things to keep in mind?

One of the main things is to really look inside yourself and question yourself. Ask yourself really important questions like,

@heartsinbloodhealth

As you said, you recently recovered from COVID-19. What was that experience like for you personally and professionally?

In the beginning of the year, we actually started seeing patients have lung flues and pneumonias with weird symptoms and they were just really sick in January. I believe that COVID-19 was definitely here in the United States in California in January. But we didnt know what it was yet. But then when they started announcing it in February, we definitely got more traffic in the office and we were taking care of patients - even though it was primary and oncology, a lot of the patients in the beginning stages when they were starting to get sick would come to us. So, we were being exposed to COVID-19 and it spread through the staff.

The COVID-19 symptoms started out mild for me. I just figured it was the stress from working and being the only medical assistant since we were short-staffed during that time. Then once the rest of the clinic tested positive, I was like, Oh, no! I think I may have it!

I got tested. The week after I tested positive the illness became super severe for me. I have asthma and type-one diabetes - it went straight into my lungs. I decided to just face it and not be crippled by fear. I set my mind to believe that I would not die from it. Because Id learned with my chronic illness that you just have to stay positive no matter how grim it looks. And really just focus on what you can do to heal.

During the time when it was really severe, I went to the hospital and towards the end, I was able to go home and recover.

Now, its been three months since I've been cleared. I still have lung damage. My body is still healing. My body is still recovering from it in that aspect. But I was very fortunate because Ive had over ten years of experience being a chronic illness patient and working in the medical field. With that experience in mind, I knew how to take care of my immune system and listen to my body. When I got it, it was the beginning of the pandemic when doctors really didnt know anything about it. They didnt know what to tell me. They didnt know how to help me. So, for a lot of it I was on my own. I had to figure out how to survive COVID-19.

Luckily, my knowledge and prayer and by the grace of God and blessings, I was able to fight it, survive it. I was able to apply my knowledge to quickly do a regimen of trying to boost my immune system fast. I made a point to not stress, not think negatively, not fear, and not feed into any of that. I just tried to stay calm through the whole thing. Eventually, after 70 days, I was cleared.

What was the most difficult symptom of COVID-19 for you?

I think, honestly, the worst part was not being able to breathe. Even now, its still hard to breathe. It felt like I was suffocating 24-7, there was no relief. I was even on a breathing machine, not a ventilator, but a nebulizer breathing machine every four hours and taking medication just to be able to survive. I probably should have gone on a ventilator at that point because my oxygen stats were just in the 80s and so low and I was so sick. But I just didnt want to go on the ventilator. When I went into the hospital, they gave me fluids, took all the tests to make sure there was no other organ damage.

So, it was just kind of traumatic, honestly! But I learned a lot. Im grateful to be alive. Im grateful to be able to help anyone, you know? Or to face it without as much fear, even though it is really scary. To have hope and to try to stay calm.

You seem connected with people, both in-person and digitally. How has this helped, how has Instagram helped spread your story?

Its so amazing because I just started sharing my story of being a chronic illness patient and so many people could relate to that. Then when I started sharing my story and my experiences working in the medical field so many more people would message me and connect with me. Ive met so many amazing people, its amazing whats come out of Instagram. Whats come out of this community is just so many different opportunities and abilities to be able to hear other peoples stories, share my story, be able to learn from others, be able to help others. Its made me a better person and its also helped me be able to have a stronger voice.

When youre about to start your day, what is the final thing that passes through your mind before you open the doors to work?

Every morning, I just say a prayer, walking in, in my mind. And I dont know if this is going to sound corny but because this is my calling, I really, truly want to make the most of my job.

I pray right before I walk in every single morning that God uses me as a light of warm light and love to each person that I come across so that my day is very purposeful and meaningful and that Im able to uplift someone in some way. Or help someone in another way or just comfort a patient in a way that they need. I pray every day that my light shines through to the patients and staff around me.

Still have specific questions about becoming a medical assistant? Read our ultimate guide to becoming a Medical Assistant now.

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This Is Why I'm Working as a Medical Assistant While I Advance My Education - Nurse.org