As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...

The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...

The FDA will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, ...

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

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

Sarepta Therapeutics Inc. has refused to pause all shipments of its Elevidys treatment after three deaths were linked to the ...

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 is planning to ask Sarepta Therapeutics to voluntarily stop all shipments of its gene ...

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

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...

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

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