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

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

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

Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), ...

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

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

The boys live in Reed City with their mother Tabatha; father Michael, who is the Osceola County Road Commission manager and ...

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support ...

Donnacha O’Neill is a happy and outgoing two and a half year old little boy from Meath who loves to be outside and is due to ...

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

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 ...

Sarepta Therapeutics (NASDAQ:SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...