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

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

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its ...

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.

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

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

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

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

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