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

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

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

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

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

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

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

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

NEW YORK – Carmine and Vincent Gagliano, two brothers from Brooklyn, were living the dream on Thursday at Yankee Stadium. It was like watching two kids in the candy store. They love the Yankees that ...

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

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

Background Over the last few years, there has been increasing attention to the involvement of the central nervous system in Duchenne muscular dystrophy (DMD). The aim of this study was to assess the ...