BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on sevasemten, an ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

Sarepta’s blockbuster gene therapy has come under scrutiny following a string of patient deaths.

Additional analyses from the givinostat clinical development program review observations on weight-based flexible dosing, characterization of thrombocytopenia, correlations between pharmacodynamic ...

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

Wedbush initiated coverage on Solid Biosciences, Inc. (NASDAQ:SLDB), a gene therapy company focused on treating Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Pratteln, Switzerland, January 15, 2026 – Santhera Pharmaceuticals (SIX: SANN) announces that the Swiss Agency for Therapeutic Products (Swissmedic), has approved AGAMREE® (vamorolone) for the ...

Please provide your email address to receive an email when new articles are posted on . New therapies for muscular dystrophy must go beyond traditional corticosteroid administration. Treatments that ...

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. Muscular dystrophy, as a category, is quite ...

– Company to host an Industry Forum to discuss the lived experience of Becker and clinical advancements featuring a leading neuromuscular disease expert and a patient advocate – Edgewise is sponsoring ...