Globally, Duchenne is the most prevalent form of muscular dystrophy in children. It primarily affects boys, and it is estimated to occur in approximately one in every 3,500 to 5,000 live male births ...

On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular dystrophy: A second patient who had been treated with Elevidys has died.

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD). WVE ...

Capricor’s Biologics License Application (BLA) for Deramiocel in DMD remains under priority review, with a PDUFA target action date of August 31, 2025. About Duchenne and Becker Muscular Dystrophy ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support licensureAdvisory Committee meeting scheduled for July 30, 2025Mid-cycle ...

Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running ...

By Upsher-Smith Laboratories, LLC Jun 16, 2025 Updated Jun 16, 2025 ...

Becker Muscular Dystrophy, much like Duchenne Muscular Dystrophy, is a type of genetic disorder that causes progressive muscle weakness and degeneration over time, primarily in males.

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.

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. The biotech drugmaker said Sunday it was ...