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 ...

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 ...

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

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

Duchenne 型肌营养不良症（Duchenne muscular dystrophy，DMD）是一种常见的儿童肌肉疾病，由 DMD 基因突变导致肌肉中肌营养不良蛋白（dystrophin）产生异常。 这不仅会引发肌肉问题，还常伴有脑部相关共病，如智力障碍（约 22% 的患者）、自闭症（约 6%）和注意力缺陷障碍（约 18%）等。

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

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