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 ...

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

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

The ouster of the FDA's chief regulator of cell and gene therapies came immediately after a disagreement with her boss over a ...

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

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 ...

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

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

The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...