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), ...

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

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

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

Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...

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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...

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

Sarepta Therapeutics (NASDAQ:SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

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

The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...