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Category Archives: Muscular Dystrophy Treatment

CANbridge Pharmaceuticals Enters into Duchenne Muscular Dystrophy Gene Therapy Research Agreement with University of Washington School of Medicine -…

Posted: Published on November 5th, 2021

BEIJING & CAMBRIDGE, Mass., November 01, 2021--(BUSINESS WIRE)--CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that it has entered into a two-year sponsored research agreement with the University of Washington School of Medicine, in Seattle, Washington, for gene therapy research in Duchenne muscular dystrophy (DMD), a rare neuromuscular disease. The program will be under the direction of Jeffrey Chamberlain, Ph.D., professor in the Departments of Neurology, Medicine and Biochemistry, the McCaw Endowed Chair in Muscular Dystrophy at the University of Washington School of Medicine, and Director of the Senator Paul D Continue reading

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Elizabeth M. McNally, M.D., Ph.D., FAHA, named the American Heart Association’s 2021 Distinguished Scientist in Basic Cardiovascular Sciences -…

Posted: Published on November 5th, 2021

Embargoed until 7 a.m. CT / 8 a.m Continue reading

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Fulcrum Therapeutics Reports Recent Business Highlights and Third Quarter 2021 Financial Results – The Bakersfield Californian

Posted: Published on November 5th, 2021

On-track to initiate enrollment by year-end in Phase 1b clinical trial of FTX-6058 in people with sickle cell disease Losmapimod update planned for Q1 2022 Raised $144.2 million in gross proceeds from August 2021 public offering, extendingcash runway into 2024 Conference call scheduled for 8:00 a.m. ET today CAMBRIDGE, Mass., Nov. 04, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. Continue reading

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Why Sarepta Therapeutics Shares Dropped More Than 16% This Week – Motley Fool

Posted: Published on November 5th, 2021

Key Points Shares in Sarepeta Therapeutics (NASDAQ:SRPT) fell more than 16% this week, according to data from S&P Global Market Intelligence. The stock closed last Friday at $448.68 then fell to as low as $376.26 on Wednesday, a day after the company reported earnings Continue reading

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Dying patients with rare diseases struggle to get experimental therapies – The Detroit News

Posted: Published on July 18th, 2021

Christina Bennett | Kaiser Health News At 15, Autumn Fuernisen is dying. She was diagnosed at age 11 with a rare degenerative brain disorder that has no known cure or way to slow it down: juvenile-onset Huntingtons disease Continue reading

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Analysis: The FDA Is a Melting Iceberg – GovExec.com

Posted: Published on July 18th, 2021

The byzantine world of pharmaceutical regulation has recently broken into the public consciousness, causing a bit of a panic. Aducanumabthe first new Alzheimers treatment in nearly two decadeswas approved by the Food and Drug Administration on June 7 despitescantevidence of benefit, and against thenearly unanimousadvice of the agencys expert advisers. Op-eds called the decision, which could triggerbillions of dollarsin new government spending, a false hope, bad medicine, and a new low. Continue reading

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Neuromuscular Disorders Treatment Market: Rise in awareness about genetic disorders is expected to drive the market – BioSpace

Posted: Published on February 2nd, 2021

Neuromuscular Disorders Treatment Market: Overview Neuromuscular disorders encompass several diseases, divided in terms of localization into disorders that involve cranial and spinal motor neurons, peripheral nerves, nerve plexuses, spinal nerve roots, neuromuscular junctions, and/or muscles. Neuromuscular disorders affect the nerves that control voluntary muscles. When the neurons become unhealthy or die, communication between the nervous system and muscles breaks down Continue reading

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The Duchenne Muscular Dystrophy Market to witness 1.5X growth in the next decade Jumbo News – Jumbo News

Posted: Published on February 2nd, 2021

As per the report by Persistence Market Research (PMR), the globalduchenne muscular dystrophy marketis expected to experience strong growth. The market is also estimated to expand at a CAGR of 48.3% during the forecast period. The global duchenne muscular dystrophy market is estimated to bring in US$ 10,664.5 million revenue by the end of 2026. Continue reading

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Bayer: Transforming Healthcare through Collaborations and Breakthrough Innovation – Taiwan Business TOPICS – Taiwan Business TOPICS

Posted: Published on February 2nd, 2021

Finding effective solutions to the worlds health issues requires novel, holistic approaches and collaborative ingenuity. Healthcare companies must therefore increasingly shift their focus to the research and development of medical technologies, biopharmaceuticals, and innovative therapies to treat conditions and diseases that are difficult or nearly impossible to tackle using traditional medicines. Such a reality is well understood by Bayer AG Continue reading

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Orchard Therapeutics Appoints Braden Parker as Chief Commercial OfficerCommercial Launch of Libmeldy in Europe On-Track for 1H 2021 – BioSpace

Posted: Published on February 2nd, 2021

BOSTONandLONDON, Jan. 29, 2021 (GLOBE NEWSWIRE) --Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced the appointment of Braden Parker to the role of Chief Commercial Officer (CCO). In this capacity, Mr. Continue reading

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