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

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and ...

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

To date, over 900 Duchenne patients have been treated with delandistrogene moxeparvovec. "The signal for ALF is exceptionally ...

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

Shares of Sarepta Therapeutics have plunged again after the 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 ...

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

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

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 ...

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