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

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 ...

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

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

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

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

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

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

The crisis over Sarepta Therapeutics' Duchenne therapy offers a valuable lesson: Listening to critics, and responding with ...

After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy to ambulatory people but maintain a halt it implemented June 15 for ...

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