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), ...

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 ...

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

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

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

JAR of Hope helps support a team of researchers and doctors who are aiming to eliminate Duchenne muscular dystrophy – a fatal ...

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support licensure Advisory Committee meeting scheduled for July 30, 2025 Mid-cycle ...

Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dystrophy care, targeting broader ages and mutations. We are witnessing the fastest pivot in rare-disease ...