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

The move follows the death of two teenagers who were administered the treatment, as well as the death of a 51-year-old ...

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

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...

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

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

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 ...

The U.S. Food and Drug Administration is planning to ask Sarepta Therapeutics to voluntarily stop all shipments of its gene ...

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

The restructuring enacted by Sarepta Therapeutics is expected to save up to $400 million in annual costs. Read why I'm ...

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