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

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

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

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

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

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 will request Sarepta Therapeutics to voluntarily stop all shipments of its gene therapy ...

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

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...