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

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 ...

Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an unrelated drug.

In light of recent patient deaths, the FDA has also revoked its platform designation for Sarepta’s AAVrh74 technology. The ...

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

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

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 ...

Elevidys is a gene therapy approved to treat Duchenne muscular dystrophy. Last month, Sarapta halted sales of the therapy for non-ambulatory patients.

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

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 ...