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

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

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

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

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

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

NEW YORK – Carmine and Vincent Gagliano, two brothers from Brooklyn, were living the dream on Thursday at Yankee Stadium. It ...

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

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...

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