Category Archives: Muscular Dystrophy Treatment

For Dr. Continue reading →

A Phase 1 clinical trial evaluating Edgewise Therapeutics experimental oral therapy EDG-5506 in healthy volunteers and men with Becker muscular dystrophy (BMD) has dosed the first BMD patients. The study (NCT04585464) is currently recruiting up to eight men, ages 18 to 55, with BMD at its single site in San Antonio, Texas; more information can be foundhere. Top-line data from these patients are expected by the end of the year Continue reading →

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 →

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

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 →

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 →

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 →

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 →

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 →

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 →