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, ...

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

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 ...

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

Sarepta Therapeutics to lay off 493 workers, including 80 in Ohio, amid FDA probe, stock plunge, and concerns over Duchenne ...

The U.S. Food and Drug Administration will request Sarepta Therapeutics to voluntarily stop all shipments of its gene therapy ...

Shares of Sarepta Therapeutics (NASDAQ: SRPT) plunged 26.85% in Friday pre-market after a clinical trial participant died ...