He was diagnosed with Duchenne muscular dystrophy at age 5.Family remembers the day everything changed"I can remember the day ...

To date, over 900 Duchenne patients have been treated with delandistrogene moxeparvovec. "The signal for ALF is exceptionally ...

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s ...

Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in ...

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

Background Over the last few years, there has been increasing attention to the involvement of the central nervous system in Duchenne muscular dystrophy (DMD). The aim of this study was to assess the ...

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...