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

Sarepta Therapeutics has rejected a call by the Food and Drug Administration to stop shipments of its muscular dystrophy drug ...

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

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

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 ...

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

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.

A Cambridge-based company developing gene therapies for rare diseases is laying off more than a third of its workforce.

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

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

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