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

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

The move follows the death of two teenagers who were administered the treatment, as well as the death of a 51-year-old ...

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

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

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 ...

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 ...

Sarepta (SRPT) plans to continue Elevidys shipments for Duchenne muscular dystrophy, despite FDA request not to do so after ...

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 ...