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

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

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

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

Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene ...

Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain ...

"There were tears everywhere," Hana Herrick, school services coordinator at Lurie Children’s Hospital, tells PEOPLE ...

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 ...

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

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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...

Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...