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

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 ...

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

Capricor shares four-year data showing Deramiocel slowed DMD progression and preserved function, with maintained safety in long-term use.

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), ...

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

Anthony Lukken has Duchenne's Muscular Dystrophy, which causes a person's muscles to become weaker over time.

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

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