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

The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...

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

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

A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of the FDA.

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...

As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm ...

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

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

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...

Sarepta Therapeutics said it will continue shipping its gene therapy Elevidys for Duchenne muscular dystrophy patients despite an FDA request to pause distribution following multiple patient deaths.

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle ...