At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

The death will likely further stoke safety concerns around Sarepta's gene therapies, as well as raise questions around the ...

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

A third patient has died this year after getting a gene therapy from Sarepta Therapeutics Inc., raising questions about the ...

The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute ...

According to multiple reports, a 51-year-old man with limb-girdle muscular dystrophy died from acute liver failure after ...

Second patient death from liver failure after Sarepta's Elevidys gene therapy triggers FDA investigation. Stock crashes 41% ...

A third fatality has been recorded among patients receiving treatment with a Sarepta gene therapy, adding to the troubles ...

The Purdy family shares their insights on navigating their child’s rare neurodegenerative disease and their direct ...

Sarepta Therapeutics (SRPT) said a third patient has died from acute liver failure after receiving one of its gene therapies, ...

In a surprise, a FDA advisory panel voted that risks tied to a GSK blood cancer drug called Blenrep outweighed the benefits ...