The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.

A brief skirmish between Sarepta Therapeutics and the FDA has ended before escalating into a full-on regulatory clash, as the company has bowed to the agency’s demand. | A brief skirmish between ...

Sarepta Therapeutics said it has paused all shipments of its Elevidys treatment for Duchenne muscular dystrophy in the U.S.

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

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

Sarepta plummets after a third death in its muscular dystrophy program for investigational gene therapies, prompts FDA action and intensifies safety scrutiny.

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 ...

FDA issued Complete Response Letter Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial ...

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 ...