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 ...

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

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

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

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

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

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

A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...

Sarepta Therapeutics lays off 493 workers amid FDA probe, stock drop, and concerns over its gene therapy treatment, Elevidys.

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

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...