Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment ...

Interim data showed 110% average microdystrophin expression and up to 88% dystrophin-positive muscle fibers in the first three participants. Two patients saw an 8% average heart function improvement ...

Regenxbio Inc (NASDAQ:RGNX) revealed on Wednesday new interim data from two additional patients in the Phase 1/2 portion of the AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy for ...

The FDA on Friday revised the safety information for Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys, adding a boxed warning to alert patients and doctors about the risk of ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. A second person with Duchenne muscular dystrophy has ...

January 4, 2008 — A small trial testing an experimental gene therapy in 4 patients with Duchenne's muscular dystrophy shows promising results. Investigators at Leiden University Medical Center, in the ...

New York, Aug. 23, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) today announced the publication of jointly developed consensus ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. The young ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting in morning trading. The ...