CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an ...

The pharma company said it would lay off a third of its workforce and add a black-box warning to its controversial gene ...

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, ...

Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk.

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

In this week’s edition of InnovationRx, we look at Moderna’s use of quantum computers, a new top drug regulator at the FDA, ...

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

On an investor call Friday, analysts grilled the company over its apparent lack of transparency on the matter.

The restructuring enacted by Sarepta Therapeutics is expected to save up to $400 million in annual costs. Read why I'm ...

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

The company is also pausing research for several treatments it has been developing for another form of muscular dystrophy.