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Muscular Dystrophy Association Awards 15 Grants Totaling More Than $4 Million for Neuromuscular Disease Research – Stockhouse

NEW YORK , Sept. 23, 2020 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) announced today the awarding of 15 new MDA grants totaling more than $4 million toward research focused on a variety of neuromuscular diseases (NMDs), including Duchenne muscular dystrophy (DMD), Charcot-Marie-Tooth disease (CMT), Becker's muscular dystrophy (BMD), spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). This round of grant funding reinforces MDA's unwavering commitmentin the face of declining income due to the COVID-19 pandemicto the progress of neuromuscular disease research and builds on the more than $1 billion MDA has already invested in research to uncover new treatments and cures for NMDs since its inception. Some grants will go into effect this year, while others will be awarded in 2021.

"We continue to fund the most innovative research that will lead us to cures for a range of neuromuscular diseases," says Sharon Hesterlee, PhD, executive vice president and chief research officer for Muscular Dystrophy Association. "We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable."

Dr. Hesterlee added, "Although COVID led the cancellation of MDA's spring review session, we are pleased to announce the funding of these projects, which were reviewed in 2019."

The newly funded projects will aim to advance research discoveries and new therapy development in multiple areas. The awarded grants will fund studies to further advance our understanding of genetic causes of and risk factors for NMDs, investigate new approaches to developing gene therapies and other innovative potential treatments, including stopping disease progression and improving genetic testing technologies.

For a complete list of individual awards for this grant cycle, visit MDA's website and explore the Grants at a Glance section. Highlights from the grant awards for this grant cycle include:

ALS grants will be announced separately later this month, as will grants being given jointly by MDA and other organizations.

About the Muscular Dystrophy Association For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care . As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best in class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA's COVID-19 guidelines and virtual events are posted at mda.org/COVID19 . For more information, visit mda.org .

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Muscular Dystrophy Association Awards 15 Grants Totaling More Than $4 Million for Neuromuscular Disease Research - Stockhouse

Thalassemia Treatment Market projected to expand at a CAGR of 7.9% from 2018 to 2026 – The Daily Chronicle

Transparency Market Research (TMR)has published a new report titled, Thalassemia Treatment Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026.According to the report, theglobal thalassemia treatment marketwas valued at US$ 842.0 Mn in 2017 and is projected to expand at a CAGR of 7.9% from 2018 to 2026. Increase in R&D investment by key players for developing new drugs for treating thalassemia and rise in demand for chelating therapy are anticipated to fuel the growth of the global market from 2018 to 2026. Asia Pacific and Middle East & Africa are expected to dominate the global market owing to increase in prevalence of thalassemia disorder and high adoption of chelation therapy & blood transfusion for treatment by doctors as well as patients. The market in Asia Pacific is projected to expand at the fastest CAGR during the forecast period. Growth of the market in the region is attributed to large base of private clinics and hospitals, rise in number of thalassemia population requiring chelation therapy services after spleen surgery, and surge in adoption of blood transfusion among patients. The thalassemia treatment market in Latin America is likely to expand at a moderate growth rate during the forecast period.

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Value Added Features in Thalassemia Treatments to Propel Global Market

The global thalassemia treatment market is projected to be driven by value added features offered by various thalassemia drug manufacturing companies in order to streamline the day to day work flow and increase revenue. The thalassemia treatment provides limited range of features and benefits ranging from patient pain heeling remedies to treatment procedures. For instance, very less number of people go for the much beneficial chelation therapy. These features help physicians and nurses to streamline the chelation therapy required for patients to maintain their daily workflow efficiently and effectively. Key players offering thalassemia treatment are coming up with value added features such as bone marrow transplantation, stem cell regeneration, gene editing methodologies, and effective modality features used for drug manufacturing along with creating a prominent candidate molecule for drugs. These features can reduce the overall operating cost and improve the overall effectiveness and efficiency of treatment practices. Companies are focusing on the development of combined drug therapy in their system to effectively integrate chelating therapy or other treatment procedure at an affordable cost. These value added features save time for physicians and help improve thalassemia patient survival performance.

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Chelation Therapy to be Highly Lucrative Segment

Traditionally, blood transfusion based on type of thalassemia treatment was the most commonly used procedure among thalassemia patients. This treatment type was associated with availability of donor and cost of treatment procedure. Moreover, chelation therapy based on thalassemia treatment are priced on perpetual license model and are expensive. Chelation therapy treatment enables patients to practice intensive therapy to treat acute iron overload leading to 90% recovery among thalassemia patients. These chelation therapy based treatments address specific challenges faced during the treatment procedure. The chelation therapy treatment facilitates benefits such as pain relief, and increase in motion of blood flow among patients.

Asia Pacific Presents Significant Opportunities

North America and Europe accounted for major share of the global thalassemia treatment market in 2017 and are likely to gain market shares by 2026. High rate of immigration from tropical regions, increasing health care budgets by governments, and government initiatives to promote thalassemia treatment technique contributed to the leading share of these regions. Asia Pacific is projected to be the most attractive market for thalassemia treatment, with highest attractiveness index. The market in Asia Pacific is expected to expand at a high CAGR of 9% during the forecast period due to large number of thalassemia patients opting for chelation therapy in developing countries such as India and China. Well-established health care facilities, medical tourism for treatment of thalassemia, and high adoption of blood transfusion safety technique in countries such as Turkey and GCC Countries are likely to drive the market in Middle East & Africa. The market in Latin America is poised to expand at a moderate growth rate during the forecast period.

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Trend of R&D among Leading Players to Increase Geographic Presence

The report also provides profiles of leading players operating in the global thalassemia treatment market. bluebird bio, Inc., Acceleron Pharma, Inc., Novartis AG, Celgene Corporation, and Shire plc (Takeda Pharmaceuticals) are the leading players operating in the global market. Companies operating in the thalassemia treatment market aim to increase geographic presence and research & development through strategic acquisitions and collaborations with leading players in respective domains and region. In December 2017, Shire plc committed to pay approximately US$ 1,409.9 Mn to contract vendors for administering and executing clinical trials. Other prominent players operating in the global thalassemia treatment include Incyte Corporation, Kiadis Pharma, Gamida Cell, Celgene Corporation, and Bellicum Pharmaceuticals.

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Meet the 2020 finalists for the $1 million Sanford Lorraine Cross Award honoring innovation in medical science – PRNewswire

SIOUX FALLS, S.D., Sept. 29, 2020 /PRNewswire/ -- Sanford Health, the largest provider of rural healthcare in the country, today announced the finalists for the biennial Sanford Lorraine Cross Award which honors life-changing breakthroughs and innovations in medical science.

The 2020 finalists are:

Mark Denison, MD Director of Pediatric Infectious Diseases Principle Investigator (Denison Lab) Departments of Pediatrics and Pathology, Vanderbilt University

Carl June, MD Director of the Center for Cellular Immunotherapies Perelman School of Medicine, University of Pennsylvania

Michael Welsh, MD Investigator (Howard Hughes Medical Institute) Director (Pappajohn Biomedical Institute) Carver College of Medicine, University of Iowa

Sanford Health is the only health system in the country to award a $1 million prize for achievements in the medical sciences. A winner will be selected at a special ceremony in Sioux Falls, South Dakota on Tuesday, December 8, 2020.

While traditional awards in the medical sciences focus on the significance of the contribution of a researcher or clinician, the Sanford Lorraine Cross Award celebrates the role that the award candidate has played in bringing a new emerging transformative medical innovation across the finish line to patients, and their efforts in overcoming challenges, forging collaborations, and ensuring a successful outcome.

Finalists for the Sanford Lorraine Cross Award are determined through a rigorous selection process that uses machine learning to identify innovative areas of discovery and breakthrough science. The candidate selection is then focused on the areas with the most promising transformative potential for patients. An interdisciplinary scientific advisory board narrowed down the top areas of innovation. The individuals who have made the greatest contribution in these three areas are then identified as the finalists.

"Sanford Health has a long history of relentlessly challenging the status quo to improve health care," said Kelby Krabbenhoft, CEO, Sanford Health. "The Sanford Lorraine Cross Award honors someone pioneering that change with a medical breakthrough, innovation or treatment to transform global health. It starts with a $1 million prize, and it continues with a life-changing impact for us all."

The award is named after the Cross of Lorraine, a global symbol first chosen as emblematic for those who took action against disease in 1902 at the International Tuberculosis Congress in Berlin. In recent history, Sanford Health has used it to symbolize profound innovation and progress in the medical sciences and also includes the image in its own institutional logo.

The Sanford Lorraine Cross Award is supported in perpetuity through an endowment established by donors to the Sanford Health Foundation. Donors who invest $1 million or more are honored as members of the Founders Circle, a distinguished group celebrated for its visionary generosity. These individuals are united by their passion for creating a better tomorrow, inspiring future generations of courageous innovators, and leaving a legacy of health and healing.

The December awards event will be livestreamed on Sanford Health's social media channels on Twitter, Facebook and YouTube, and regularly covered on Sanford Health News.

Here's a closer look at the 2020 Lorraine Cross finalists:

Mark Denison, MD

Dr. Mark Denison of Vanderbilt University has studied coronaviruses since the 1980s. In 2007, his lab discovered that coronaviruses have a protein that acts as a powerful proofreader during replication, meaning that the virus can self-correct errors in its RNA sequence.

This makes coronaviruses difficult to treat, but Dr. Denison and his team theorized that if they could slip past the proofreaders, they could prevent the virus from growing. Through a partnership with Gilead Sciences, they discovered Remdesivir, a drug that can evade the proofreading system and terminate the growth, thus killing the virus.

Though Remdesivir was shelved after failed attempts to treat hepatitis and Ebola, it has experienced a rebirth during today's COVID-19 pandemic and is undergoing clinical trials for treatment.

Since the spark of COVID-19, Dr. Denison's story is the world's story. His work on Remdesivir has received coverage from The New Yorker, CNN, The New York Timesand others.

Without his early discovery of this proofreading mechanism, it is unlikely scientists would have been able to jump start therapeutic solutions for the current COVID-19 pandemic.

Carl June, MD

T cells are like the body's security guards, recognizing harmful intruders and triggering an immune response. Dr. Carl June of the University of Pennsylvania, working with colleague Dr. Bruce Levine, was able to reprogram selected T cells to recognize and destroy leukemia cancer cells.

This therapy is now named KYMRIAH by Novartis and is FDA-approved to treat acute lymphoblastic leukemia (ALL).

In 2010, June and Levine treated their first patient a retired 65-year-old corrections officer named Bill Ludwig. After just a few infusions, Ludwig fell severely ill and spent a week in the ICU. Then, all of the sudden, he woke up and found the masses had disappeared.

A second patient, Emily Whitehead, had relapsed twice at the age of seven with an extremely aggressive form of leukemia. Like Ludwig, Whitehead got severely ill as the therapy tried to rid her of her cancer. However, this time Dr. June recalled an arthritis drug (from his daughter's struggle with juvenile arthritis) that could manage her side effects.

The story of Emma Whitehead was featured in a 2012 article in the New York Timesand covered in Forbes, PARENTSMagazine and the PBS documentary "Cancer: The Emperor of All Maladies."

Michael Welsh, MD

Dr. Michael Welsh isa pulmonary physician who wanted to know how human airways work, particularly howsalt ions moveacross the airway wall. This led him to study cystic fibrosis (CF),an inherited disease that causes lifelong vulnerability todestructive lunginfections and an early death.

He discovered that the CFTR gene instructs the cell to make a channelfor chloride.This was a monumentalbreakthroughbecause it linked the genetics and the lung infections.Importantly, it also clearly identified thetarget that needed repair.

He then discovered ways that different inherited mutations in the CFTRgene disrupt the chloride channel.Thatwork providedthe blueprint that has continued to guide therapeuticdevelopment.He demonstrated that thecommon CF mutation could becorrected in the lab, a discovery that ignited CF drugdevelopment and provided the confidence, knowledge and tools thatensured success.His workhas been widely covered, including in theThe New York Times.

The excitingadvances arising from Dr. Welsh's research have dramatically changed the livesof people withCF.Today, 90%of CF patients have ahighly effective therapy.His discoverieswere instrumental in arecent finding, published with colleaguesin the March 2019 journalNature, that an approved drug normally used totreat fungal infections could also do the job of theCFTR channel.While additional treatmentswill also be developed, the fundamental properties required to treat the defectremain those defined by the pioneering work of Welsh.

About Sanford Health Sanford Health, one of the largest health systems inthe United States, is dedicated to the integrated delivery of health care, genomic medicine, senior care and services, global clinics, research and affordable insurance. Headquartered inSioux Falls, South Dakota, the organization includes 46 hospitals, 1,400 physicians and more than 200 Good Samaritan Society senior care locations in 26 states and 10 countries. Learn more about Sanford Health's transformative work to improve the human condition atsanfordhealth.orgorSanford Health News.

Media Contact: Angela Dejene [emailprotected] 218-280-0148

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Meet the 2020 finalists for the $1 million Sanford Lorraine Cross Award honoring innovation in medical science - PRNewswire

Genomics Market to Rise at 19.5% CAGR and Reach USD 82.60 Billion by 2027; Advances in Diagnostic Applications will Lead to a Wider Product Adoption,…

September 29, 2020 08:39 ET | Source: Fortune Business Insights

Pune, Sept. 29, 2020 (GLOBE NEWSWIRE) -- The global genomics market size is projected to reach USD 82.60 billion by the end of 2027. The massive investments in the research and development of efficient products will have a massive impact on the growth of the market in the coming years.

According to a report published by Fortune Business Insights, titled Genomics Market Size, Share & COVID-19 Impact Analysis, By Type (Products (Instruments & Software and Consumables) and Services), By Technology (Polymerase Chain Reaction (PCR), Next Generation Sequencing (NGS), Microarray, Sanger Sequencing, and Others), By Application (Diagnostics, Research, and Others), By End User (Research Institutes, Healthcare Facilities & Diagnostic Centers, Pharmaceutical & Biotechnological Companies, Contract Research Organization (CROs)), and Regional Forecast, 2020-2027, the market was worth USD 18.85 billion in 2019 and will exhibit a CAGR of 19.5% during the forecast period 2020-2027.

Genomics is a concept that involves the human gene and sequencing for treatment and study of several diseases or potential diseases. The study of human genetics helps identify and track critical diseases, with equal focus on the qualities and behaviour of a specific individual. In the past few years, genomics has contributed to the growth of numerous similar branches including the popular precision medicine.

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The massive investments in the research and development of genomics and genomic sequencing will have a positive impact on the growth of the overall market in the coming years. The presence of several large scale companies has had a massive impact on the growth of the market in recent years and the influx of start-ups will create opportunities for growth in the foreseeable future.

Recent Clinical Studies have Indicated the Potential of Genomics to Tracking Patterns of Covid-19 Spread

The recent coronavirus outbreak has created a sense of panic among businesses across the world. Although healthcare industry has witnessed a contrasting impact, as compared to a few other sectors, there has been a recent surge in the need for healthcare professionals. The lack of skilled labour is consequential to the hesitancy among workers due to the severity of the disease. Although there are several ongoing research activities associated with the treatment of the coronavirus, there has been little success in this field.

Accounting to increasing cases of Covid-19 across the world, researchers are focusing on implementing preventive and precautionary measures through newer concepts. The excessive research associated with the use of genomics in Covid-19 pandemic has yielded a few successful measures. As a result, genomics has recently attracted attention from across the world.

The whole world is fighting the novel coronavirus. Sectors and industries are devasted due to the major loss caused by COVID-19 in business. The authorities of several countries have initiated lockdown to prevent the spread of this deadly virus. Such plans have caused disturbances in the production and supply chain. But, with time and resolution, we will be able to combat this stern time and get back to normality. Our well-revised reports will help companies to receive in-depth information about the present scenario of every market so that you can adopt the necessary strategies accordingly.

To get to know more about the short-term and long-term impacts of COVID-19 on this market, please visit: https://www.fortunebusinessinsights.com/industry-reports/genomics-market-100941

Constant Product Innovations are Consequential to Increasing R&D Efforts

The report encompasses several factors that have contributed to the growth of the overall market in recent years. Among all factors, the increasing number of product innovations, driven by rising investments in the research and development of the product, has made the highest impact on the growth of the market. In April 2020, Eurofins announced the launch of a new product in the genome sequencing space.

The company introduced SARS-CoV 2 full length genome sequencing, a product that is categorized under the next-generation (NGS) sequencing category. Increasing number of such product innovations will have a positive impact on the growth of the overall market in the coming years.

North America to Emerge Dominant; Increasing Activities associated with Whole-genome Sequencing will Emerge in Favor of Market Growth

The report analyses the ongoing market trends across five major regions, including North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. Among all regions, the market in North America is projected to emerge dominant in the coming years. The increasing research activities associated with whole-genome sequencing by private companies as well as government organizations will contribute to the growth of the regional market.

As of 2019, the market in North America was worth USD 8.27 billion and this value is projected to rise at a considerable pace in the coming years. The market in Asia Pacific will derive growth form the efforts put in towards genetic sequencing by countries such as China.

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List of companies profiled in the Genomics Market report:

Industry Developments:

February 2020: Nebula Genomic announced the launch of a new product that will offer whole genome sequencing at US$ 299 to its customers.

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Next-Generation Sequencing (NGS) Market Share and Global Trend By Type (Products, Instruments & Software, Consumables, Services), By Application (Diagnostics, Research), By End User (Research Institutes, Healthcare Facilities & Diagnostic Centres, Pharmaceutical & Biotechnological Companies, Contract Research Organization) & Geography Forecast till 2026

Molecular Diagnostics Market Share and Global Trend By Product Type (Instruments Reagents & Consumables), Application (Infectious Disease, Blood Screening, Histology & Oncology), Technique (Hospitals Amplification, Hybridization & Sequencing Techniques), End User (Hospitals, Clinical & Pathology Labs) and Geography Forecast till 2025

Polymerase Chain Reaction (PCR) Market Share & Industry Analysis, By Type (Standard PCR, Real-time PCR, and Digital PCR), By Product (Instruments and Reagents & Consumables), By Indication (Infectious Diseases, Oncology, Genetic Disorders, and Others), By End User (Hospitals & Clinics, Pharmaceutical & Biotechnology Industries, Diagnostic Centers, and Academic & Research Organizations), and Regional Forecast, 2019-2026

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Genomics Market to Rise at 19.5% CAGR and Reach USD 82.60 Billion by 2027; Advances in Diagnostic Applications will Lead to a Wider Product Adoption,...