He was diagnosed with Duchenne muscular dystrophy at age 5. "I can remember the day he got diagnosed," said his older sister, ...

Four-year data show preservation of cardiac function, including LVEFSkeletal muscle disease progression continues to slow ...

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

A North Texas family is sharing a son's journey with Duchenne muscular dystrophy, a rare and progressive muscle-wasting ...

The death of a boy with Duchenne muscular dystrophy, tied to a gene therapy, is above all a tragedy. But it is also ...

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

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

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

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

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

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