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Stem Cell Therapy Market Revenue, Key Players, Supply-Demand, Investment Feasibility and Forecast 2026 – The Think Curiouser

October 23rd, 2020 2:57 pm

Global Stem Cell Therapy Market Research Assessment and Forecast 2020 2028 offers an assessment of the global Stem Cell Therapy market and a forecast of market demand by type and segment of end users. This study offers a comprehensive industry assessment as it includes statistically validated and validated industry market data, facts, critical findings, and historical data. It also contains predictions made using an appropriate set of methodologies to obtain accurate values. Market analysis offers information according to the categories identified through market segmentation, which includes product type, geography and applications.

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Provides a worldwide analysis of Stem Cell Therapy market growth for both regional and global markets, including an understanding of business strategy, development trends, opportunities and development status of important regions.

The study provides quantitative information on key industry trends, market size (value and volume) and development of each end-user segment of Stem Cell Therapy, import / export of Stem Cell Therapy by region, production volume and main country. Historical and future consumption trends, as well as market innovations, as well as the challenges faced by major Stem Cell Therapy manufacturers as well as end users, are suggested.

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Each market participant included in the Stem Cell Therapy market study is ranked according to its manufacturing coverage, market share, existing and new launches, ongoing R&D projects and business strategies. In addition, market research for Stem Cell Therapy provides an assessment of strengths, weaknesses, opportunities and threats (SWOT). The report assesses and explores the prospects for the global platelet market, including sales, production and use, as well as historical data and projections.

Stem Cell Therapy Market Segmentation

Type Analysis of Stem Cell Therapy Market:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCsBone Marrow-Derived Mesenchymal SCsEmbryonic SCsOther Sources

Applications Analysis of Stem Cell Therapy Market:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal DisordersWounds & InjuriesCardiovascular DiseasesGastrointestinal DiseasesImmune System DiseasesOther Applications

Market research monitors the global situation and explains how the industry will create promising profit margins for manufacturers in the post-COVID-19 crisis. The market research aims to offer further information on the economic downturn, the latest scenario and the impact of COVID-19 on the industry as a whole.

Scope of the global Stem Cell Therapy Market Report:

This research covers detailed manufacturer data such as shipment, price, gross margin, interview record, revenue, business distribution, etc. This data helps the user to get to know the competition better. The report also includes all regions and countries of the world, showing the complex status of regional growth such as market size, volume and value, and price data.

Likewise, the study also covers segment data, including segment type, market segment, channel segment, and so on, which contains different market segment sizes, both in value and volume. In addition, it covers consumer data from different industries, which is very important for service providers.

Analyzing the Investment Potential of the Global Stem Cell Therapy Market Report

The report hovers across the past and current dynamics to deduce significant developments in the aforementioned market, thus effectively encouraging agile business outcome The report also is a ready-to-refer documentation that entails substantial information featuring the developments across segments and their role in growth optimization Systematic R&D activities and concomitant resource planning are thoroughly touched upon in this report featuring the development graph in global Stem Cell Therapy market. The report also ensures investor participation towards directing manufacturer and vendor activities in a bid to achieve significant competitive edge. Market based developments are also accurately sectioned in both value-based volume-based calculations to thoroughly encourage reader understanding and subsequent growth potential in global Stem Cell Therapy market.

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Stem Cell Therapy Market Revenue, Key Players, Supply-Demand, Investment Feasibility and Forecast 2026 - The Think Curiouser

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Potential impact of Covid-19 on Rheumatoid Arthritis Stem Cell Therapy Market Growth and Demand, Concludes Fact.MR – The Cloud Tribune

October 23rd, 2020 2:57 pm

The globalRheumatoid Arthritis Stem Cell Therapy marketstudy presents an all in all compilation of the historical, current and future outlook of the market as well as the factors responsible for such a growth. With SWOT analysis, the business study highlights the strengths, weaknesses, opportunities and threats of each Rheumatoid Arthritis Stem Cell Therapy market player in a comprehensive way. Further, the Rheumatoid Arthritis Stem Cell Therapy market report emphasizes the adoption pattern of the Rheumatoid Arthritis Stem Cell Therapy across various industries.Request Sample Reporthttps://www.factmr.com/connectus/sample?flag=S&rep_id=1001The Rheumatoid Arthritis Stem Cell Therapy market report highlights the following players:The global market for rheumatoid arthritis stem cell therapy is highly fragmented. Examples of some of the key players operating in the global rheumatoid arthritis stem cell therapy market include Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc, International Stem Cell Corporation, TiGenix and others.

The Rheumatoid Arthritis Stem Cell Therapy market report examines the operating pattern of each player new product launches, partnerships, and acquisitions has been examined in detail.Important regions covered in the Rheumatoid Arthritis Stem Cell Therapy market report include:

North America (U.S., Canada)Latin America (Mexico, Brazil)Western Europe (Germany, Italy, U.K., Spain, France, Nordic countries, BENELUX)Eastern Europe (Russia, Poland, Rest Of Eastern Europe)Asia Pacific Excluding Japan (China, India, Australia & New Zealand)JapanMiddle East and Africa (GCC, S. Africa, Rest Of MEA)

The Rheumatoid Arthritis Stem Cell Therapy market report takes into consideration the following segments by treatment type:

Allogeneic Mesenchymal stem cellsBone marrow TransplantAdipose Tissue Stem Cells

The Rheumatoid Arthritis Stem Cell Therapy market report contain the following distribution channel:

HospitalsAmbulatory Surgical CentersSpecialty ClinicsHave Any Query? Ask our Industry Experts-https://www.factmr.com/connectus/sample?flag=AE&rep_id=1001

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The Rheumatoid Arthritis Stem Cell Therapy market report offers a plethora of insights which include:

Changing consumption pattern among individuals globally.Historical and future progress of the global Rheumatoid Arthritis Stem Cell Therapy market.Region-wise and country-wise segmentation of the Rheumatoid Arthritis Stem Cell Therapy market to understand the revenue, and growth lookout in these areas.Accurate Year-on-Year growth of the global Rheumatoid Arthritis Stem Cell Therapy market.Important trends, including proprietary technologies, ecological conservation, and globalization affecting the global Rheumatoid Arthritis Stem Cell Therapy market.

The Rheumatoid Arthritis Stem Cell Therapy market report answers important questions which include:

Which regulatory authorities have granted approval to the application of Rheumatoid Arthritis Stem Cell Therapy in Health industry?How will the global Rheumatoid Arthritis Stem Cell Therapy market grow over the forecast period?Which end use industry is set to become the leading consumer of Rheumatoid Arthritis Stem Cell Therapy by 2028?What manufacturing techniques are involved in the production of the Rheumatoid Arthritis Stem Cell Therapy?Which regions are the Rheumatoid Arthritis Stem Cell Therapy market players targeting to channelize their production portfolio?Get Full Access of the Report @https://www.factmr.com/report/1001/rheumatoid-arthritis-stem-cell-therapy-market

Pertinent aspects this study on the Rheumatoid Arthritis Stem Cell Therapy market tries to answer exhaustively are:

What is the forecast size (revenue/volumes) of the most lucrative regional market? What is the share of the dominant product/technology segment in the Rheumatoid Arthritis Stem Cell Therapy market? What regions are likely to witness sizable investments in research and development funding? What are Covid 19 implication on Rheumatoid Arthritis Stem Cell Therapy market and learn how businesses can respond, manage and mitigate the risks? Which countries will be the next destination for industry leaders in order to tap new revenue streams? Which new regulations might cause disruption in industry sentiments in near future? Which is the share of the dominant end user? Which region is expected to rise at the most dominant growth rate? Which technologies will have massive impact of new avenues in the Rheumatoid Arthritis Stem Cell Therapy market? Which key end-use industry trends are expected to shape the growth prospects of the Rheumatoid Arthritis Stem Cell Therapy market? What factors will promote new entrants in the Rheumatoid Arthritis Stem Cell Therapy market? What is the degree of fragmentation in the Rheumatoid Arthritis Stem Cell Therapy market, and will it increase in coming years?Why Choose Fact.MR?

Fact.MR follows a multi- disciplinary approach to extract information about various industries. Our analysts perform thorough primary and secondary research to gather data associated with the market. With modern industrial and digitalization tools, we provide avant-garde business ideas to our clients. We address clients living in across parts of the world with our 24/7 service availability.

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Potential impact of Covid-19 on Rheumatoid Arthritis Stem Cell Therapy Market Growth and Demand, Concludes Fact.MR - The Cloud Tribune

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Lucrative Opportunities in North America to Propel the Growth of the Amniotic Fluid Stem Cell Therapy Market 2018 2026 – TechnoWeekly

October 23rd, 2020 2:57 pm

Amniotic Fluid Stem Cell Therapy Market 2018: Global Industry Insights by Global Players, Regional Segmentation, Growth, Applications, Major Drivers, Value and Foreseen till 2024

The report provides both quantitative and qualitative information of global Amniotic Fluid Stem Cell Therapy market for period of 2018 to 2025. As per the analysis provided in the report, the global market of Amniotic Fluid Stem Cell Therapy is estimated to growth at a CAGR of _% during the forecast period 2018 to 2025 and is expected to rise to USD _ million/billion by the end of year 2025. In the year 2016, the global Amniotic Fluid Stem Cell Therapy market was valued at USD _ million/billion.

This research report based on Amniotic Fluid Stem Cell Therapy market and available with Market Study Report includes latest and upcoming industry trends in addition to the global spectrum of the Amniotic Fluid Stem Cell Therapy market that includes numerous regions. Likewise, the report also expands on intricate details pertaining to contributions by key players, demand and supply analysis as well as market share growth of the Amniotic Fluid Stem Cell Therapy industry.

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Amniotic Fluid Stem Cell Therapy Market Overview:

The Research projects that the Amniotic Fluid Stem Cell Therapy market size will grow from in 2018 to by 2024, at an estimated CAGR of XX%. The base year considered for the study is 2018, and the market size is projected from 2018 to 2024.

Leading manufacturers of Amniotic Fluid Stem Cell Therapy Market:

key players operating in global amniotic fluid stem cell therapy market are Stem Shot, Provia Laboratories LLC, Thermo Fisher Scientific Inc. Mesoblast Ltd., Roslin Cells, Regeneus Ltd. etc. among others.

The report covers exhaustive analysis on:

Regional analysis includes

Report Highlights:

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Some important highlights from the report include:

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/23101

The Questions Answered by Amniotic Fluid Stem Cell Therapy Market Report:

And Many More.

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Lucrative Opportunities in North America to Propel the Growth of the Amniotic Fluid Stem Cell Therapy Market 2018 2026 - TechnoWeekly

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Global Adipose Derived Stem Cell Therapy Market 2020 Demand, Business Growing Strategies, Industry Segmentation and Forecast 2025 – TechnoWeekly

October 23rd, 2020 2:57 pm

An up-to-date intelligence study by Global Adipose Derived Stem Cell Therapy Market Growth (Status and Outlook) 2020-2025 provides the present scenario of the market and offers a comparative assessment of the market. The report provides historical data, significance, statistical data, size & share, market price & demand, business overview, market analysis by product and market trends by key players. It detects that the global Adipose Derived Stem Cell Therapy market by technological advancements and the presence of a large number of players, who are making the competitive landscape distributed. The report further highlights current growth factors, market threats, attentive opinions, and competitive analysis of major Adipose Derived Stem Cell Therapy market players, value chain analysis, and future roadmap.

Competitiveness:

The Adipose Derived Stem Cell Therapy market is fragmented and is characterized by the presence of key vendors and other prominent vendors. Key vendors are trying to maintain themselves in the global Adipose Derived Stem Cell Therapy market, whereas, regional vendors are focusing on product offerings to establish themselves in the market. Vendors are providing a different range of product lines intensifying the competitive scenario.

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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.

Market Potential:

The market vendors have been forecasted to obtain new opportunities as there has been an increased emphasis on spending more on the work of research and development by many of the manufacturing companies. Additionally, many of the market participants are anticipated to make a foray into the emerging economies that are yet to be explored so as to find new opportunities. The global Adipose Derived Stem Cell Therapy market has gone through rapid business transformation by good customer relationships, drastic and competitive growth, significant changes within the market, and technological advancement in the worldwide market.

Topmost list manufacturers/ key player/ economy by business leaders leading players of the market are: AlloCure, Tissue Genesis, Antria, Cellleris, Corestem, Celgene Corporation, Mesoblast, Cytori Therapeutics, Pluristem Therapeutics, Intrexon, Lonza, BioRestorative Therapies, Pluristem Therapeutics, iXCells Biotechnologies, Cyagen, Celltex Therapeutics Corporation,

Geographically, this report studies market share and growth opportunity in the following key regions: Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia), Middle East & Africa (Egypt, South Africa, Israel, Turkey, GCC Countries)

On the basis of product segment, this report covers: Autologous Stem Cells, Allogeneic Stem Cells,

On the basis of Application segment, this report covers: Therapeutic Application, Research Application,

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Important Take-Away:

Moreover, the report has covered the PEST analysis by region and key economies across the globe, PORTERs analysis, and detailed SWOT analysis of key players to analyze their strategies. It guides companies to make effective business strategy decisions by knowing the global Adipose Derived Stem Cell Therapy market conditions and sentiment within the market. The report serves to adjust investment allocation by outlining key focus areas of the industry.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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Marketandresearch.biz is a leading global Market Research agency providing expert research solutions, trusted by the best. We understand the importance of knowing what global consumers watch and buy, further using the same to document our distinguished research reports. Marketandresearch.biz has worldwide presence to facilitate real market intelligence using latest methodology, best-in-class research techniques and cost-effective measures for worlds leading research professionals and agencies. We study consumers in more than 100 countries to give you the most complete view of trends and habits worldwide. Marketsandresearch.biz is a leading provider of Full-Service Research, Global Project Management, Market Research Operations and Online Panel Services.

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Global Adipose Derived Stem Cell Therapy Market 2020 Demand, Business Growing Strategies, Industry Segmentation and Forecast 2025 - TechnoWeekly

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Otilimab Well Tolerated and May Improve Synovitis in Active Rheumatoid Arthritis – Rheumatology Advisor

October 23rd, 2020 2:56 pm

Treatment with otilimab, a monoclonal antibody that inhibits granulocyte-macrophage colony-stimulating factor (GM-CSF), is well tolerated, despite optimal exposure, and may improve synovitis in patients with active rheumatoid arthritis (RA), according to study results published in Lancet Rheumatology.

Previous studies have reported that GM-CSF can act as a proinflammatory cytokine and has an important role in a broad range of immune-mediated conditions, making it a valuable therapeutic target in patients with RA. The objective of the current mechanistic phase 2a study was to determine the effect of otilimab on the GM-CSF-chemokine (C-C motif) ligand 17 (CCL17) axis and synovitis in patients with RA.

The multicenter, randomized, placebo-controlled study (ClinicalTrials.gov Identifier: NCT02799472) included adult patients with RA from across 9 sites in the United States, Poland, and Germany. Participants were randomly assigned to receive either 180 mg of subcutaneous otilimab or placebo. The primary outcome was change over time in 112 biomarkers. Secondary end points included change from baseline in synovitis, osteitis, and erosion assessed by the RA magnetic resonance imaging (MRI) Scoring (RAMRIS) system, RA MRI Quantitative (RAMRIQ) score, and safety evaluation.

A total of 39 patients with RA who were randomly assigned to receive otilimab (n=28) or placebo (n=11) were included in the final cohort. Weekly subcutaneous injections of otilimab 180 mg or placebo were administered for 5 weeks, then every other week until week 10, followed by a 12-week safety follow-up.

Results showed that mean serum concentrations of the GM-CSF-otilimab complex peaked at week 4 (138.4 ng/L), but reduced from week 6 to 12, with CCL17 concentrations decreasing and then returning to baseline at week 12 (least-squares mean ratios at weeks 2, 4, 6, and 8 were 0.65, 0.68, 0.78, and 0.68, respectively). In the placebo group, no significant changes in CCL17 concentrations were noted. There were also no differences between groups for all other biomarkers.

Results from imaging measures of synovitis showed a reduction from baseline to week 12 in patients who received otilimab. Differences in MRI outcomes were minimal, and a trend for reduced synovitis and osteitis was seen early during active treatment, but not at 12 weeks after the treatment was stopped. The RAMRIS synovitis score showed a least-squares mean change from baseline of -1.3 (SE, 0.6) in the otilimab group and a mean change of 0.8 (SE, 1.2) in the placebo group (P =.11). The RAMRIQ synovitis score showed a least-squares mean change from baseline of -1417.0 L in the otilimab group and -912.3 L with placebo (P =.75).

All adverse events were reported to be mild or moderate, and the number of adverse events was similar in the otilimab and placebo groups (39% and 36%, respectively). The most common adverse event was cough (7%) in the active treatment group and pain in extremity (18%) and RA (18%) in the placebo group. There were no serious adverse events or deaths.

The study had several limitations, including the relatively small sample size, imbalanced baseline disease-modifying antirheumatic drug use among treatment groups, and the determination of the RAMRIS synovitis score based on assessment by a single radiologist.

The findings of this study support the rationale for the further clinical development of otilimab as a treatment option for patients with [RA]. Additionally, the effect of otilimab on CCL17 indicates that CCL17 shows promise as a pharmacodynamic biomarker for otilimab in future studies, the researchers concluded.

Disclosure: This clinical trial was supported by GlaxoSmithKline. Please see the original reference for a full list of authors disclosures.

Genovese MC, Berkowitz M, Conaghan PG, et al. MRI of the joint and evaluation of the granulocytemacrophage colony-stimulating factorCCL17 axis in patients with rheumatoid arthritis receiving otilimab: a phase 2a randomised mechanistic study. Lancet Rheumatol. Published online October 7, 2020. doi:10.1016/s2665-9913(20)30224-1

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Otilimab Well Tolerated and May Improve Synovitis in Active Rheumatoid Arthritis - Rheumatology Advisor

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Comparison of the effectiveness of pilates exercises, aerobic exercises, and pilates with aerobic exercises in patients with rheumatoid arthritis -…

October 23rd, 2020 2:56 pm

Background:Rheumatoid arthritis (RA) is a rheumatic disease characterized by erosive synovitis and polyarthritis. Exercise is known to improve many symptoms in RA patients.

Aim:This study was designed to compare the effects of pilates exercises, aerobic exercises, and combined training including pilates with aerobic exercises on fatigue, depression, aerobic capacity, pain, sleep quality, and quality of life.

Methods:Thirty voluntary RA patients were included in this study. Patients were divided into three groups equally, and treatment was applied to each group for 8 weeks. Pilates exercises were practiced to the first group, aerobic exercises were practiced to the second group, and combined training was performed to the third group. Fatigue, depression, aerobic capacity, pain, sleep quality, and quality of life were evaluated using Fatigue Severity Scale (FSS), Beck Depression Inventory (BDI), 6-minute walk test (6MWT), McGill Pain Questionnaire- Short Form (MPQ-SF), Pittsburg Sleep Quality Index (PSQI), and Rheumatoid Arthritis Quality of Life (RAQoL), respectively.

Results:The results of the present study showed significant improvements for the first group on fatigue, depression, aerobic capacity, and quality of life (p < 0.05). Improvements in all parameters except for pain were obtained for the second and third groups (p < 0.05). In addition, there was no statistically significant difference among the treatment groups in assessments (p > 0.05).

Conclusion:Pilates exercises may have similar effects to aerobic exercises in patients with RA. Addition of clinical pilates exercises to the routine treatment of RA may enhance the success of rehabilitation. Trial registrationNCT03836820.

Keywords:Aerobic exercises; Fatigue; Pilates exercises; Rheumatoid arthritis.

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Comparison of the effectiveness of pilates exercises, aerobic exercises, and pilates with aerobic exercises in patients with rheumatoid arthritis -...

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ZetrOZ Receives Funding from the National Institute of Health to Find New Ways to Reduce Arthritic Pain for Over 60 Million Americans Using…

October 23rd, 2020 2:56 pm

TRUMBULL, Conn., Oct. 22, 2020 /PRNewswire/ --ZetrOZ Systems, developers of sustained acoustic medicine (SAM), an FDA-cleared home-use ultrasound device that helps reduce pain associated with tendon, ligament, and muscle injuries, has received funding from the National Institute of Health (NIH) to find a way to reduce arthritic pain utilizing non-surgical, non-narcotic technology.

Arthritic pain has been primarily managed by nonsteroidal anti-inflammatory drugs (NSAIDs), namely diclofenac. Diclofenac, which is administered topically, is limited in its efficacy due to its limited ability to penetrate the skin. New findings from researchers at the University of Cincinnati show SAM technology's ability to deliver NSAIDs locally and effectively, demonstrating 380% enhanced delivery compared to existing technology for the treatment of joint pain.

"Targeted and localized drug delivery is the holy grail for many therapeutic agents, and this has been demonstrated with small-molecule therapeutics, combined with our latest sam 2.0," says Dr. George Lewis, CEO of ZetrOZ Systems. "The research community is excited by the findings, as it has broad implications for the local delivery of therapeutic agents without the need for oral delivery."

The study published in the Journal of Therapeutic Delivery Vol 11. NO 6.investigated the use of sam 2.0, "a multi-hour, wearable, localized, sonophoresis transdermal drug delivery device for the penetration enhancement of diclofenac through the skin." The use of sam 2.0 medical technology resulted in increased delivery of diclofenac by 380% and increased depth of penetration of the skin by 32%. Findings concluded that sustained acoustic medicine can be used as an effective transdermal drug-delivery device for nonsteroidal anti-inflammatory drugs.

"Our collaborations with the top research institutions such as the University of Cincinnati foster continuous innovation on sustained acoustic medicine in the treatment of chronic pain," adds Lewis.

About ZetrOZ Systems

ZetrOZ Systems is an FDA cGMP and ISO 13585 medical technology company headquartered in the southern coastal region of Connecticut. The organization also has manufacturing facilities across the United States. ZetrOZ Systems produced UltrOZ, samSport and samPro 2.0 to provide safe and effective treatment options for prevalent conditions such as arthritis. Learn more at zetroz.comand samrecover.com.

Media Contact: [emailprotected]

Related Images

sam-2-0-medical-technology.jpg SAM 2.0 medical technology featured in the Journal of Therapeutic Delivery for the treatment of chronic arthritis pain

SOURCE ZetrOZ Systems

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ZetrOZ Receives Funding from the National Institute of Health to Find New Ways to Reduce Arthritic Pain for Over 60 Million Americans Using...

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Rheumatoid arthritis drug tocilizumab falls short as treatment for hospitalized Covid-19 patients in three studies – msnNOW

October 23rd, 2020 2:56 pm

CHANDAN KHANNA/AFP/Getty Images A medic prepares to transfer a patient on a stretcher from an ambulance outside of Emergency at Coral Gables Hospital where Coronavirus patients are treated in Coral Gables near Miami, on July 30, 2020. - Florida has emerged as a major new epicenter of the US battle against the disease, with confirmed cases recently surpassing New York and now second only to California. The state toll has leapt over the past week and more than 6,500 people have died from the disease there, according to health officials. More than 460,000 people have been infected with the virus in Florida, which has a population of 21 million, and a quarter of the state's cases are in Miami. The US has tallied a total of 151,826 deaths from COVID-19, making it the hardest-hit country in the world. (Photo by CHANDAN KHANNA / AFP) (Photo by CHANDAN KHANNA/AFP via Getty Images)

Tocilizumab, a repurposed rheumatoid arthritis drug once considered a promising treatment for hospitalized patients with Covid-19, generally did not increase patients' chances of survival or help them get better faster, according to three trials published this week.

However, a fourth trial did find the drug increased the chances of survival, but because it was an observational study, the results are considered less definitive.

Three of the trials were published Tuesday in the Journal of the American Medical Association and the other was published Wednesday in the New England Journal of Medicine.

While this leaves the picture for tocilizumab use a bit muddy, the studies taken together show the drug isn't a magic bullet that should be used in all hospitalized patients with Covid-19, but they leave the door open for possible use in specific patient groups.

"It is possible that forthcoming results from other randomized trials will help us identify specific groups of people who will benefit. However, growing evidence supports current guidelines, which recommend against tocilizumab use outside of clinical trials," Dr. Jonathan Parr told CNN.

Parr, an assistant professor in the division of infectious diseases at the University of North Carolina at Chapel Hill, wrote an editorial that was published alongside the three studies in JAMA.

Tocilizumab, sold under the brand name Actemra and used to treat rheumatoid arthritis, blocks interleukin-6, a molecule that produces inflammation.

Earlier in the pandemic, the drug became widely used in the United States after reports from China and Europe appeared to show it helped very ill patients who experienced a so-called cytokine storm by extinguishing the out-of-control inflammation that developed in response to the coronavirus.

However, those early reports were mostly observational, meaning they took existing data and analyzed it as opposed to designing a trial specifically to assess tocilizumab.

But now the results from trials designed to look at the drug prospectively are beginning to come in.

The first of the three JAMA studies found that hospitalized patients in Italy who were not yet in intensive care and who received tocilizumab fared no better than those who received standard care. The trial was stopped early due to "futility."

A similar study in France found that tocilizumab may have led to slight improvements by day 14 over usual care, but there were no differences in survival by day 28.

The third study found that the risk of in-hospital death was about 30% lower in US patients receiving tocilizumab within the first two days of ICU admission compared to those who didn't get the drug.

But because it was an observational trial there could be factors that affected the results other than how well the drug itself worked.

"We specifically studied tocilizumab administration in very sick patients, all of whom required ICU level of care. In contrast, the [other two trials] studied patients with much milder illness severity," said Dr. Shruti Gupta of Brigham and Women's Hospital in Boston, who led the study team.

Dr. David Leaf, the trial's senior author, added, "We focused on early use of tocilizumab -- within the first two days of ICU admission. This may be the key to tocilizumab's efficacy -- administering it prior to the occurrence of irreversible organ injury," he said in an email.

Gupta said their findings need confirmation by a large randomized, controlled trial.

The fourth study, published Wednesday in the New England Journal of Medicine, used the gold standard of trial design-- it's a randomized, double-blind, placebo control trial.

It enrolled 243 patients from seven Boston-area hospitals who were admitted with Covid-19 at the height of the surge in that region.

"The primary goal of the trial was to determine if tocilizumab, administered intravenously at a moderate stage of the patients' disease, could prevent progression to intubation or death," said Dr. John H. Stone, lead study author and director of Clinical Rheumatology at Massachusetts General Hospital, told CNN via email.

But the findings were not encouraging for the use of tocilizumab.

"Our data do not support the concept that early IL-6 receptor blockade is an effective treatment strategy in moderately ill patients hospitalized with Covid-19 infection," said Stone.

Results show that the chances of intubation or death were about similar in both groups and patients in both groups essentially took the same amount of time to discontinue supplemental oxygen.

So, where does that leave tocilizumab?

"Tocilizumab may still have a role in COVID-19. Several large trials are expected to come out soon that will tell us more about how and when it should be used, if at all," said Parr, noting that it "shouldn't be taken off the table completely, but we need more convincing evidence before using it routinely."

Stone agreed that the drug may still benefit other patient groups, but he stressed the importance of doing these trials.

"An important take-away point from our trial and the overall experience with IL-6 receptor blockade is that any such approach to the treatment of COVID-19 must be subjected to randomized, blinded trials," he said, adding that the trials should be done "before the adoption of widespread use."

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Rheumatoid arthritis drug tocilizumab falls short as treatment for hospitalized Covid-19 patients in three studies - msnNOW

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Rheumatoid Arthritis Stem Cell Therapy Market Highlights On Future Development 2018 to 2028 – TechnoWeekly

October 23rd, 2020 2:56 pm

New Study Reports Rheumatoid Arthritis Stem Cell Therapy Market 2020 Global Market Opportunities, Challenges, Strategies and Forecasts 2026 has been Added on Fact.MR.OverviewStarting from the fundamental details, the report provides a complete overview of the industry along with a proper market profile. The details provided here about the crucial technologies used for manufacturing and product management purpose makes it easier to have a thorough insight into the Global Rheumatoid Arthritis Stem Cell Therapy Market. Based on the information obtained, the market has been segmented into various categories. It predicts the growth rate of the Global Rheumatoid Arthritis Stem Cell Therapy Market in between the forecasted period, having a base year as 2020.

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This report focuses Global Rheumatoid Arthritis Stem Cell Therapy market, it covers details as following:Competitive landscape

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Market DynamicsThe report identifies all the key aspects that drive the super-fast growth of the international Global Rheumatoid Arthritis Stem Cell Therapy Market. In this context, it identifies the crucial aspects regarding the pricing part of the concerned product. It analyses the market value of each of the products and services as well in the report, including the various kinds of volume trends. Prime aspects that are covered in this report range from the effect of growing population at international level, accelerating technological growth, and the analysis of level of demand and supply as evident in the Global Rheumatoid Arthritis Stem Cell Therapy Market. The report also covers extensive studies regarding various effects in relation to the initiatives taken by the government and the competitive platform that is there in the Global Rheumatoid Arthritis Stem Cell Therapy Market in between the forecasted period. Segmental AnalysisThe report does thorough segmentation of international Global Rheumatoid Arthritis Stem Cell Therapy Market upon taking various factors associated with the growth of the market. It does a thorough regional segmentation. These segmentation based studies are done with an intention of achieving a thorough and specific insight of the Global Rheumatoid Arthritis Stem Cell Therapy Market. The report does a regional analysis of the key zones of the world, starting from the US, North America, Latin America, Middle East & Africa, and Asia. Modes of researchThe research being done by experienced experts has done a comprehensive analysis of Global Rheumatoid Arthritis Stem Cell Therapy Market based on Porters Five Force Model, taking the assessment period between 2020-2026 into account. Additionally, a deep SWOT analysis is done to facilitate quick decision making for the associated people in the Global Rheumatoid Arthritis Stem Cell Therapy Market.

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Report covers:Comprehensive research methodology of Global Rheumatoid Arthritis Stem Cell Therapy Market.This report also includes detailed and extensive market overview with gap analysis, historical analysis & key analyst insights.An exhaustive analysis of macro and micro factors influencing the market guided by key recommendations.Analysis of regional regulations and other government policies impacting the Global Rheumatoid Arthritis Stem Cell Therapy Market.Insights about market determinants which are stimulating the Global Rheumatoid Arthritis Stem Cell Therapy Market.Detailed and extensive market segments with regional distribution of forecasted revenuesExtensive profiles and recent developments of market players

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Platelet glycoprotein Ib alpha chain as a putative therapeutic target for juvenile idiopathic arthritis: a Mendelian randomization study – DocWire…

October 23rd, 2020 2:56 pm

Objective:To ascertain the role of platelet glycoprotein Ib alpha chain (GPIb) plasma protein levels in cardiovascular, autoimmune and autoinflammatory diseases and whether its effects are mediated by platelet count.

Methods:We performed a two-sample Mendelian randomization (MR) study, using both cis and trans-acting protein expression quantitative trait loci (pQTL) near GP1BA and BRAP genes as instruments. To assess if platelet count mediated the effect, we then performed a two-step MR study. Putative associations (GPIb/ platelet count/ disease) detected by MR analyses were subsequently assessed using multiple-trait-colocalization analyses.

Results:After correcting for multiple testing (P 210-3), GPIb, instrumented by either cis-pQTL or trans-pQTL, was causally implicated with increased risk of juvenile idiopathic arthritis (JIA oligoarticular and rheumatoid factor negative subtypes). These effects of GPIb appear to be mediated by platelet count and are supported by strong evidence of colocalization (probability of all three traits sharing a common causal variant 0.80). GPIb instrumented by cis-pQTL did not appear to affect cardiovascular risk, although the GPIb trans-pQTL associates with increased risk of cardiovascular diseases and autoimmune diseases but decreased risk of autoinflammatory diseases, suggesting this trans- instrument acts through other pathways.

Conclusion:The role of platelets in thrombosis is well-established, however our findings provided some novel genetic evidence that platelets may be causally implicated in the development of JIA, and GPIba as a putative therapeutic target for these JIA subtypes.

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Try these three natural remedies to manage arthritis pain – Germiston City News

October 23rd, 2020 2:56 pm

Arthritis affects over 350 million people in the world.

It is also a leading cause of disability and can affect mobility and interrupt peoples ability to perform simple daily tasks.

World Arthritis Day was on October 12, which aimed to raise awareness about rheumatic and musculoskeletal diseases. Although strides have been made to find suitable treatments, many continue to suffer from debilitating pain.

Owner and founder of The Harvest Table, Catherine Clark, said there are natural remedies which can help manage symptoms associated with arthritis.

There is no cure for arthritis, but if you support your body with the right foods and supplements, you can alleviate some of the pain so that it doesnt become a hindrance in your daily life, said Clark.

Clark added that arthritis can affect ones energy levels, cause pain, and is a direct result of a loss of collagen in the bones.

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The key is to find solutions that will help you feel less fatigued, while also managing pain and replenishing the collagen lost, she said. Clark offers three natural remedies to manage arthritis pain:

Anti-inflammatory foodsArthritis fatigue is real, and according to the Arthritis Foundation, unchecked inflammation and pain largely contribute to your energy levels, along with certain medications that can cause drowsiness.Boosting your energy starts with nourishing your body with the right foods, especially those with high anti-inflammatory properties that help your bodies repair process. This will not only alleviate pain but will reduce the inflammation in your body. To effectively manage your arthritis, reduce the amounts of processed foods and saturated fats as these will only further contribute to your symptoms. Instead, choose fresh fruits and vegetables, especially green vegetables and berries. Also include fish and nuts, which both contain high anti-inflammatory properties.

Slow and gentle movementMovement is a critical part of recovery when addressing the symptoms of arthritis as it retains the suppleness of your joints. Various low impact movements can specifically tackle flexibility, strength and generally support your joints to prevent injury. Prolonged lack of movement can lead to chronic stiffness that results into joint immobility which will impact your ability to complete daily activities. Yoga is an effective solution as it reduces joint pain and also eases stress, tension and promotes better quality sleep.

Collagen-rich supplementsCollagen consists of protein building blocks, otherwise known as amino acids, which aid in cushioning our joints. When you have arthritis, this cushioning diminishes which then affects your cartilage and leads to your bones rubbing against each other without protection. Supplements like Bone Broth and Collagen granules help replenish the collagen content in your body. Bone Broth is a natural anti-inflammatory, so when you have it as part of your diet, you benefit in more ways than one. Collagen granules can help reduce both osteoarthritis and rheumatoid arthritis joint pain, improve flexibility, and helps form new bones. Although all the collagen you ingest does not go straight to your bones, increasing your intake makes them readily available for your body tissues.

Natural remedies are meant to support your body so that you can better manage pain and other symptoms associated with arthritis.

The idea is to implement small and manageable changes that contribute to you feeling better and having the energy and ability to get through the day without pain getting in the way, Clark concludes.

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Septic Arthritis Treatment Market 2020| Know The Latest Trends, Business Outlook, New Opportunities By Top Key Player | Affluence Market Reports -…

October 23rd, 2020 2:56 pm

The report on Septic Arthritis Treatment Marketgives an analysis of Septic Arthritis Treatment Industry dependent on angles that are significant for the market study. Factors like production, the share of the overall industry, revenue rate, regions and key players characterize an overall market study. This report gives an overview of market esteemed and its development in the coming years. It likewise predicts CAGR.

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The report is a thorough examination of this market over the world. It offers an outline of the market including its definition, applications, key drivers, key market players, key segments, and assembling innovation. Furthermore, the research report presents factual information on the status of the market and subsequently is an important wellspring of direction for companies and individuals interested in the businesses. Also, in-depth knowledge on the company profiles, product particulars, capacity, production worth, and market shares of key players are introduced in the report.

Major Key Players in this Septic Arthritis Treatment Market Report are as follows:

Lupin Pharmaceuticals (US), F. Hoffmann-La Roche Ltd (Swtizerland), AbbVie Inc (US), Amgen Inc (US), Bristol-Myers Squibb Company (US) , Johnson & Johnson (US), Pfizer (US) ,

The Report is Segmented as Following:

With an overall methodology for information aggregation, the market situations include significant players, cost and pricing working in particular geographies. Statistical looking over-utilized are SWOT Analysis, PESTLE analysis, prescient examination, and constant examination. Charts are plainly used to help the data format for a clear understanding of facts and figures.

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The report further explains on the restraining elements in the Septic Arthritis Treatment market for individuals, strategists, and partners to deliberately execute their techniques and accomplish their objectives. Whats more, the Septic Arthritis Treatment market is segmented based on product, innovation, and end-user. This Septic Arthritis Treatment Research report segments are altogether concentrated to offer key data like open doors for business owners, planners, and marketing staff. It encourages them to deal with their activities and execute unequivocal to earn more profits. Septic Arthritis Treatment Report offers insights on each segment and sub-segment for assisting producers to identify key opportunities and grow their business.

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Precision NanoSystems Receives $18.2 Million from the Government of Canada to Develop an RNA Vaccine for COVID-19 – Canada NewsWire

October 23rd, 2020 2:54 pm

VANCOUVER, BC, Oct. 23, 2020 /CNW/ -Precision Nanosystems, Inc. (PNI), a global leader in technologies and solutions in genetic medicine, announced today that it has received a commitment of up to $18.2 million in support from the Government of Canada under the Innovation, Science and Economic Development's (ISED) Strategic Innovation Fund (SIF) to develop a COVID-19 vaccine. PNI will use the investment to advance a best-in-class COVID-19 mRNA vaccine candidate to clinical trials.

PNI provides over 250 industry and academic partners with solutions for the development of vaccines, gene therapies, and cell therapies, in the areas of infectious diseases, oncology and rare diseases. With this investment from the Government of Canada, PNI's Chief Scientific Officer, Dr. Andrew Geall, and his team will use their state-of-the-art technology platforms and expertise in self-amplifying mRNA vectors, lipid-based drug delivery systems and nanomedicine manufacturing to develop a cost-effective COVID-19 vaccine.

As part of Canada's efforts to combat COVID-19, the Strategic Innovation Fund is working diligently to support projects led by the private sector for COVID-19 related vaccine and therapy clinical trials to advance Canada's medical countermeasures in the fight against COVID-19. "An effective vaccine will be critical as we work to contain the COVID-19 virus and prevent future infections.Today's contribution will support PNI to advance the development of a mRNA vaccine candidate through pre-clinical studies and clinical trials to help protect Canadians," stated the Honourable Navdeep Bains, Minister of Innovation, Science and Industry.

Bringing together its proprietary technology platforms, key partnerships and unparalleled expertise in nanomedicines, PNI is excited to be leading the development of a Made-in-Canada COVID vaccine. James Taylor, CEO and co-founder of PNI said "Since its inception PNI has executed on its mission to accelerate the creation of transformative medicines. It is an honour to be supported by the Canadian government in this global fight against COVID-19 and to further build capabilities for rapid response against COVID-19 and future pandemics"

About Precision NanoSystems Inc. (PNI)

PNI is a global leader in ushering in the next wave of genetic medicines in infectious diseases, cancer and rare diseases. We work with the world's leading drug developers to understand disease and create the therapeutics and vaccines that will define the future of medicine.PNI offers proprietary technology platforms and comprehensive expertise to enable researchers to translate disease biology insights into non-viral genetic medicines.

SOURCE Precision Nanosystems

For further information: Jane Alleva, Global Marketing Manager, Precision NanoSystems, Phone: 1 888 618 0031, ext 140, mobile 1 778 877 5473

http://www.precisionnanosystems.com

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Precision NanoSystems Receives $18.2 Million from the Government of Canada to Develop an RNA Vaccine for COVID-19 - Canada NewsWire

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Be Biopharma, AavantiBio Launch With Millions in Financing to Support Therapeutic Goals – BioSpace

October 23rd, 2020 2:54 pm

Its a day of firsts, with the launch of two new Cambridge, Mass.-based life sciences companies, Be Biopharma, with a focus on B cell malignancies, and AavantiBio, a gene therapy company aimed at treating rare genetic diseases.

AavantiBio launched with a $107 million Series A financing round, which includes not only a $15 million equity investment from Sarepta Therapeutics, but also an experienced executive in Alexander Bo Cumbo to helm the startup. The companys lead asset is a gene therapy treatment for Friedreichs Ataxia (FA), a rare inherited genetic disease that causes cardiac and central nervous system dysfunction.

AavantiBios gene therapy builds on the work of its co-founders, renowned gene therapy researchers Barry Byrne and Manuela Corti, who have researched FA and other genetic disorders. In addition to the foundational work of Byrne and Corti, the startup will also benefit from strategic partnerships with the University of Floridas renowned Powell Gene Therapy Center and the MDA Care Center at UF Health where Byrne and Corti maintain their research and clinical practices.

Cumbo, who spent eight years at Sarepta as chief commercial officer, will serve as the first chief executive officer of AavantiBio. He said his time at Sarepta has been incredibly rewarding as that company emerged as a pioneer in treating Duchenne muscular dystrophy and limb-girdle muscular dystrophy patients and ultimately transformed into a genetic medicine leader.

It has been a privilege to contribute to this growth and play a role in serving these communities. As I look ahead to the bright future of AavantiBio and the exciting opportunity to lead this innovative company, this same dedication to serving unmet patient needs and to leveraging deep scientific expertise will be core to our mission. I am also thrilled to continue to collaborate with the talented team at Sarepta, said Cumbo, who will continue to serve as an adviser to Sarepta through the end of 2020.

Sarepta CEO Doug Ingram praised Cumbos work over the past eight years and said he built a first-in-class rare disease commercial organization. As a partner with AavantiBio, Ingram said he looks forward to a continued relationship with Cumbo and AavantiBios efforts to advance therapies for FA and other rare diseases.

In addition to Sarepta, AavantiBios Series A was supported by Perceptive Advisors, Bain Capital Life Sciences and RA Capital Management.

Be Biopharma launched with a $52 million Series A financing round. The company will use the funds to engineer B cells to treat a range of diseases. B cells are prolific protein producers that can be collected from peripheral blood, have a programmable lifetime that could last decades, can target specific tissues, and have broad, customizable functionality.

The company intends to build on the work of co-founders David Rawlings and Richard James conducted at Seattle Childrens Research Institute. Rawlings said the goal is to build new class of engineered B cell medicines that will provide direct control over the power of humoral immunity and transform the prognosis for patients who currently have limited treatment options.

Be Biopharma is helmed by David Steinberg, a co-founder of the company and a partner at Longwood Fund, one of the supporters of the Series A.

Be Bio is capitalizing on the unique attributes of B cells to create a new category of medicine that is distinct from traditional cell or gene therapy. B cells can be engineered to express a wide variety of proteins, have the potential to generate durable responses, and can be dose-titrated and administered multiple times without the need for toxic preconditioning, Steinberg said in a statement. Moreover, the varied functions of B cells suggest that B cell medicines can address a range of conditions including autoimmune diseases, cancer, and monogenic disorders, as well as enhance the immune response to infectious pathogens. We believe Be Bio is at the forefront of a new approach to fighting disease.

In addition to Longwood Fund, the Series A financing round was supported by investment leaders Atlas Venture and RA Capital Management. Alta Partners and Takeda Ventures also supported the financing round.

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Use of Genetic Sequencing to Track SARS-CoV-2 Mutations Can Improve Diagnostic Testing Accuracy and Vaccine Effectiveness – HospiMedica

October 23rd, 2020 2:54 pm

Scientists are tracking the SARS-CoV-2 virus that causes COVID-19 by sequencing the genome of virus samples collected from diagnostic testing. Using next generation sequencing on SARS-CoV-2 will help accurately diagnose the novel coronavirus, identify mutations and track its history.

A study by scientists at the UNC School of Medicine (Chapel Hill, NC, USA) has shown how next generation genetic sequencing can track mutations in the SARS-CoV-2 virus, which can in effect help with transmission tracing, diagnostic testing accuracy and vaccine effectiveness. This type of virus monitoring is also important in diagnostic testing. Much of the testing developed to diagnose COVID-19 looks for one portion of the gene sequence that causes the novel coronavirus. If that sequence mutates, the test is no longer accurate and results will be affected.

Their recent study is the largest to focus on suburban and rural communities in which the researchers were able to reconstruct the mutational landscape of cases seen at the UNC Medical Center. Within their study, the team of scientists did find variations in the virus genetic sequence, but fortunately none of the variations were located in the portion of the virus targeted in common diagnostic testing. 175 samples from confirmed COVID-19-positive patients were analyzed, out of which 57% carried the spike D614G variant noted in similar studies. The presence of this variant is associated with a higher genome copy number and its prevalence has expanded throughout the pandemic.

The researchers will continue using NGS to track the SARS-CoV-2 virus through the remainder of 2020. The goal is to enroll every patient at UNC Hospitals with flu or respiratory symptoms for COVID-19 diagnostic testing. These samples will be sequenced and compiled to form a comprehensive profile of any virus that these patients carry, information that will continue to help a community of researchers in their fight against SARS-CoV-2 and potentially novel coronaviruses.

We are concerned about future mutations though, said Dirk Dittmer, PhD, professor of microbiology and immunology at the UNC School of Medicine, and senior author of the study. It is inherent in a virus nature to mutate. Changes in other areas of the genetic sequence can not only disrupt testing, but hinder the effectiveness of vaccines.

Because we are only looking at one gene sequence for the virus, we have told the FDA that we will continually monitor for changes in this gene sequence so that we can be assured that our test is still reliable, said Melissa Miller, PhD, director of UNC Medical Center Microbiology and Molecular Microbiology Laboratories, and a co-author of the study. NGS will help us do that.

Related Links:UNC School of Medicine

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Comprehensive Genomic Analysis in Some Patients With Breast Cancer Reveals Rare, Pathogenic TP53 Variant in Families of Ashkenazi Jewish Descent -…

October 23rd, 2020 2:54 pm

The American College of Medical Genetics currently recognizes a wide range of germline variants in the gene TP53 as pathogenic or likely pathogenic and causing the inherited disorder, Li-Fraumeni Syndrome.1 Cancers strongly associated with Li-Fraumeni syndrome include common and rare malignancies such as breast cancer, adrenocortical carcinoma, and osteosarcoma, and are normally diagnosed in patients at much younger ages than the average age of diagnosis for the general population.

In July 2020, a team of multidisciplinary researchers led by clinician-scientists at the Abramson Cancer Center at Penn Medicine, Philadelphia, added to the understanding of pathogenic TP53 variants with the publication of study in which they identified a rare, novel variant implicated in the development of Li-Fraumeni syndrome predominantly in families of Ashkenazi Jewish descent.2

Nonpathogenic p53 protein is composed of 4 major protein domains: transactivation, proline-rich, DNA-binding, and tetramerization, and is a transcription factor that activates transcription of genes encoding DNA repair machinery in healthy cells.1 Because p53 functions as a transcriptional regulator, pathogenic mutations in TP53 are most commonly observed in sequences encoding the DNA binding domain, causing the mutant p53 protein to lose its normal capacity to bind to target promoter sequences for transcriptional activation.1 Unlike more common pathogenic mutations in the DNA binding domain, the novel variant, c, 1000G>C;p. G334R, is a pathogenic missense mutation in the tetramerization domain of p53, resulting in disruption of the normal tetramerization of p53 polypeptides required to assume its native structure in wild-type carriers.2

A well-documented example of a pathogenic germline mutation in the tetramerization domain of p53 occurs in 0.3% of the general population in Southern Brazil, where a spectrum of early onset cancers was also observed in the population and confirmed as Li-Fraumeni Syndrome.3

Discovery of the novel pathogenic variant began with a research sequencing study at Abramsons Cancer Center aimed at identifying breast cancer susceptibility genes in patients with early-onset disease who were negative for germline pathogenic BRCA1/2 variants.4 Additional families with the TP53 c. 1000G>C; G334R variant were then identified from national databases and testing sites such as the National Society of Genetic Counselors ListServ and commercial genetic testing lab cases such as Ambry Genetics.

The researchers case-ascertainment database analyses resulted in a final total of 21 cases of early-onset cancer carrying the novel pathogenic TP53 variant.2 To further validate the pathogenicity of the novel variant, a team of multidisciplinary researchers utilized criteria in the 2015 ACMG/AMP Variant interpretation guidelines to evaluate predictive computational modeling of the mutant protein and in-vitro functional data of the mutant p53 in cells line.1,2

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Baby Gets Early Stem Cell Transplant to Treat Rare Disease Thanks to Newborn Screening – University of Michigan Health System News

October 23rd, 2020 2:54 pm

At birth, Minette looked perfectly healthy, and her parents took their 7 pound, 9- ounce, brown-eyed baby girl home thinking all was well.

But her newborn screening test revealed something different.

The results indicated Minette had a rare lysosomal storage disease known as mucopolysaccharidosis type I, or MPS-1. Babies usually dont show any symptoms at birth, but the condition is progressively debilitating, eventually causing permanent damage to mental development, organ function and physical abilities.

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And at nine days old in January, 2019, after a series of tests run by the newborn screen follow up team in pediatric genetics at Michigan Medicine C.S. Mott Childrens Hospital, Minette was officially diagnosed with MPS-1.

There were no signs of this disease during pregnancy or after her birth, says her mother Samantha Mejia, of West Bloomfield, Mich.

It was so important that we identified it early so she could get treatment that would give her a better chance of living a more normal life.

MPS-1 means the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules (glycosaminoglycans) within structures called lysosomes. Lysosomes are essentially the bodys recycling centers large molecules go in and come out small enough so the body can use them.

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When these molecules cant be broken down, they build up in the cell, causing many organs and tissues of the body to become enlarged, damaged and unable to work properly. Some children may develop mild to moderate mental impairment or learning difficulties, respiratory problems, sleep apnea and heart disease.

In severe cases like Minettes, children stop developing between ages 2-4, which is followed by progressive mental decline including loss of physical abilities and language skills.

MPS-1 was added to the Michigan newborn screen in August, 2017 just a little more than a year before Minette was born joining a list of more than 50 disorders that can now be detected through a simple blood test after birth.

Prior to being added to the newborn screen, many children were often diagnosed between ages one-and-a-half and three years old because they start losing developmental milestones or begin showing certain facial features as a result of glycosaminoglycans storage, such as thickened nostrils, lips or ears.

The clinical diagnosis of MPS-1 is often delayed because the symptoms tend to be non-specific early on. Newborn screening is crucial for making an early diagnosis and initiating treatment, which significantly alters the long term outcomes for patients, says Rachel Fisher, pediatric genetic counselor at Mott and a lysosomal storage disorder newborn screen coordinator for the state of Michigan.

Because of Minettes early diagnosis, her Mott care teams could quickly take next steps for treatment. She started enzyme therapy within six weeks, and at three months of age underwent four days of chemotherapy before ultimately getting a hematopoietic stem cell transplant to help replace the enzyme her body was missing.

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Global Population Sequencing Markets, 2020-2030 – Rising Adoption of Large-Scale Sequencing to Understand the Genomics of Susceptibility and…

October 23rd, 2020 2:54 pm

DUBLIN--(BUSINESS WIRE)--Oct 23, 2020--

The "Global Population Sequencing Market: Focus on Product, Method, Technology, Application, Country, and Competitive Landscape - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The population sequencing market is projected to reach $64,047.6 million by 2030 from $21,730.4 million in 2020, at a CAGR of 11.41% during the forecast period, 2020-2030.

Growth in this market is expected to be driven by the rising adoption of large-scale sequencing to understand the genomics of susceptibility and resistance from COVID-19, increasing adoption of personalized medicine for the screening and diagnosis of genetic disorders, and a global surge in direct-to-consumer genetic testing.

However, there are significant challenges that are restraining the market growth, such as lack of infrastructure to maintain, store, and share sensitive genomic data, absence of sufficient funding for the development of high-throughput genomic software tools, and poor reducibility and transability of data in clinical practice.

The market is favored by the technological advancements in the sequencing, and computational analysis solutions for a large volume of genetic data enabling a deep understanding of the genetic variants for the development of diagnostics, drug discovery, and translational research.

Furthermore, several sequencing companies are focusing on the development of high-throughput sequence platforms and polymerase chain reaction platforms, with higher sensitivity and low turn-around time to benefit the patients, enabling patient-based outcomes and implementing genomic medicine.

Within the research report, the market is segmented on the basis of product type, application, methods, and technology. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Competitive Landscape

The exponential rise in the application of precision medicine on the global level has created a buzz among companies to invest in the development of rapid diagnostics providing information on genetic mutation and optimal candidates for adjuvant chemotherapy or hormonal therapy. Due to the diverse product portfolio and intense market penetration, Illumina, Inc. has been a pioneer in this field and has been a significant competitor in this market.

The population sequencing market provided immense growth opportunities for the companies providing technology and infrastructure for large-scale health initiatives, such as Color Genomics, Inc., Helix Opco, LLC, and big data companies such as Genuity Science.

Key Questions Answered in this Report:

Key Topics Covered:

Executive Summary

1 Product Definition

1.1 Definition by Product

1.1.1 Platforms

1.1.2 Kits and Assays

1.1.3 Software Tools

1.2 Inclusion and Exclusion

1.2.1 Inclusion and Exclusion for Country-Wise Market Estimation

1.3 Scope of Work

1.4 Key Questions Answered in the Report

2 Research Methodology

3 Introduction

3.1 Market Overview

3.2 Impact of COVID-19 on Population Sequencing

3.3 Future Potential

4 Global Population Sequencing Market Dynamics

4.1 Overview

4.2 Impact Analysis

4.3 Market Drivers

4.3.1 Rising Adoption of Large-Scale Sequencing to Understand the Genomics of Susceptibility and Resistance from COVID-19

4.3.2 Increasing Adoption of Personalized Medicine for the Screening and Diagnostics of Genetic Disorders

4.3.3 Global Surge in Direct-to-Consumer (DTC) Genetic Testing

4.4 Market Restraints

4.4.1 Lack of Infrastructure to Maintain, Store and Share Sensitive Genomic Data

4.4.2 Absence of Sufficient Funding for Development of High-Throughput Genomic Software Tools

4.4.3 Poor Reducibility and Translatability of Data in Clinical Practice

4.5 Market Opportunity

4.5.1 Advancing Precision Medicine with Blockchain-Powered Artificial Intelligence

4.5.2 Technological Advancements in Sample Preparation for Population Sequencing

4.5.3 Increased Population Engagement and Data Management

5 Competitive Landscape

5.1 Key Strategies and Developments

5.1.1 Product Approval

5.1.2 Product Launches and Upgradations

5.1.3 Synergistic Activities

5.1.4 Funding and Expansion

5.1.5 Acquisitions

5.1.6 Other

6 Industry Insights

6.1 Overview

6.2 Legal Requirements and Framework in the U.S.

6.3 Legal Requirements and Framework in Europe

6.4 Legal Requirements and Framework in Asia-Pacific

6.4.1 Japan

6.5 Market Share Analysis (by Company) 2019

6.5.1 Growth Share Analysis (Opportunity Mapping)

6.5.2 By Company

7 Global Population Sequencing Initiatives (by Country)

8 Global Population Sequencing Market (by Product), $Million, 2019-2030

8.1 Introduction

8.2 Kits and Assays

8.3 Platforms

8.4 Software Tools

9 Global Population Sequencing Market (by Methods), $Million, 2019-2030

9.1 Introduction

9.2 Whole Genome Sequencing

9.3 Whole Exome Sequencing

9.4 Single-Read Sequencing

9.5 Other Sequencing Methods

10 Global Population Sequencing Market (by Technology), $Million, 2019-2030

10.1 Introduction

10.2 Polymerase Chain Reaction (PCR)

10.3 Next Generation Sequencing (NGS)

10.4 Other Technologies

11 Global Population Sequencing Market (by Application), $Million, 2019-2030

11.1 Introduction

11.2 Human health

11.2.1 Clinical Applications

11.2.1.1 Diagnostics

11.2.1.1.1 Cancer Diagnostics

11.2.1.1.2 Infectious Disease Diagnostics

11.2.1.1.3 Rare Disease Diagnostics

11.2.1.1.4 Other Diagnostics

11.2.1.2 Drug Discovery and Development

11.2.2 Translational Research Sequencing

11.3 Molecular Forensics

11.4 Blockchain in Genomics

11.4.1 Data Sharing and Monetization

11.4.2 Data Storage and Security

11.4.3 Automated Health Insurance

12 Global Population Sequencing Market (by Country), $Million, 2019-2030

13 Company Profiles

13.1 Company Overview

13.2 Role of Agilent Technologies, Inc. in Global Population Sequencing Market

13.3 Financials

13.4 Key Insights About Financial Health of the Company

13.5 SWOT Analysis

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UNC Researchers Identify Genetic Variant Linked to Drug-Induced Hypertension with Avastin – PRNewswire

October 23rd, 2020 2:54 pm

CHAPEL HILL, N.C., Oct. 22, 2020 /PRNewswire/ -- In the largest study of its kind, researchers from the University of North Carolina at Chapel Hill (UNC) have discovered a genetic variant that can be used to predict if patients will develop hypertension from the widely used cancer drug Avastin (generic name bevacizumab). The groundbreaking work is being presented this weekend by the lead researcher, Dr. Federico Innocenti, during a plenary session at the 32nd EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics. The presentation can be viewed on Sunday, October 25th at 9:45 ET on the Conference website.

VEGF inhibitors like Avastin have revolutionized cancer treatment and have been used by millions of patients. Because they target blood vessel growth and regulation, the most common and severe side effects are usually cardiovascular-related. Currently, there is no way to predict who is likely to experience these serious and potentially fatal adverse events, which can develop quickly and often require stopping or modifying treatment.

After analyzing thousands of genomic variations from over 1,000 Caucasian cancer patients across five independent clinical trials the researchers identified a genomic variant that appears twice as often in patients with hypertension than without it, and is present in over a quarter of hypertension cases. These results suggest the return on testing for this variant is very high. When assessing how many people need to be tested to avoid one severe event, the number is only 34 for Caucasian patients. If confirmed in patients of African ancestry, the number drops to just 9, given the much higher occurrence of the variant (80%+) in this population.

William R. Sellers,Professor of Medicine at the Dana-Farber Cancer Institute, who is co-chairof the Conference and not involved with the research said, "Side effects from bevacizumab can be debilitating; a simple genetic test to identify which patients will experience toxicities could help provide better and more effective treatments for our patients." Dr. Federico Innocenti, Associate Professor at UNC who led the research added, "Early identification is a potential double win. It will first help doctors identify patients at a higher risk of hypertension induced by Avastin. Then, for example, these patients can receive closer monitoring or prophylactic treatment, allowing them to continue their cancer treatment uninterrupted."

With support from the research group Emerald Lake Safety, efforts are currently underway to start a prospective study that will provide doctors with a free test to screen patients and update their treatment regimens as necessary. "Interested clinicians should contact me," says Dr. Innocenti.

Press Contact:

FedericoInnocenti, MD, PhD[emailprotected](949) 257-2074

SOURCE Federico Innocenti, MD, PhD

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UNC Researchers Identify Genetic Variant Linked to Drug-Induced Hypertension with Avastin - PRNewswire

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Getting to the Roots of Fragile X Syndrome – Tufts Now

October 23rd, 2020 2:54 pm

The cause of fragile X syndrome (FXS), the most common inherited intellectual disability, is easy to see in the lab. Under electron microscopy, an affected X chromosome exhibits a deformed tip that gives the disorder its name and pinpoints the causative gene malfunction. Theres no cure for the disease, whose symptoms include learning deficits and hyperactivity and which has been linked with autism. FXS occurs in 1 in 4,000 to 7,000 males and 1 in 8,000 to 11,000 females in the United States.

Most research on FXS has focused on the brains neurons, the cells that transmit electrical and chemical impulses. But for a decade Yongjie Yang, associate professor of neuroscience at Tufts University School of Medicine, has pursued a different path, investigating the involvement of glia cells, particularly astroglia, which support neuron function and make up more than half the brain. In the past month, hes published in the Proceedings of the National Academy of Sciences (PNAS) and Glia. Tufts Now spoke with Yang about his work.

Tufts Now: What do we know about FXS?

Yongjie Yang: Fragile X syndrome is caused by the mutation of a single gene, FMR1, that codes for the FMRP protein, which is found in all brain cells and is essential for normal cognitive development. The mutation doesnt actually change the genetic code. Instead it causes part of the gene, specifically the chemical bases CGG, to repeat. We all carry those repeats in different numbers. If you carry roughly 50 or fewer, your brain development will be normal, but if the repeats go beyond 200, you will have the full mutation and your brain will produce only 10 to 20 percent of the needed level of FMRP, especially if youre male. FXS was characterized in 1943 but the genetic mutation wasnt identified until 1991, almost half a century a later.

What is the relationship between autism spectrum disorder (ASD) and FXS?

The two are intermingled. ASD is much more common, occurring in 1 in 54 children according to new estimates. Its believed that 1 to 6 percent of people with ASD have the FXS mutation, and that mutation accounts for the largest genetic subset of those with ASD. Many people with FXS are also autistic. FXS is a learning and intellectual disability, while ASD includes a wide range of social and communications challenges.

What are the key findings of your most recent research?

The study in Glia shows that some physical symptoms of FXS can be induced in mice by eliminating FMRP from astroglia alone. So in thinking about gene therapy for FXS, we need to consider glia cells, not just neurons. Our PNAS paper is exciting because it defines a unique, distinct FMRP-dependent pathway in mouse and human astroglia that regulates communications from astroglia to neurons through mGluR5, an important receptor for glutamate, the neurotransmitter that triggers brain activity. Interestingly, this regulation pathway isnt found in neurons. Its also the first study to demonstrate how overall protein expression is changed in FMR1-deficient astroglia. Unveiling astroglia-specific molecular mechanisms involved in FXS development could give us new targets for potential therapeutics.

Whats next?

We want to better understand the pathophysiology of FXS and identify new avenues for drugs and other interventions to attenuate the effects of the disease. Of course gene therapy would be wonderful but it often takes a long, long time and carries a lot of risk. Most other studies have focused on the neuron side, and drug trials based on these studies have failed so far. Our glia/astroglia perspective gives a fresh view to search for new targets.

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Getting to the Roots of Fragile X Syndrome - Tufts Now

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