The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

Sarepta stock has tanked. Most investors don’t see the drugmaker’s executive management as credible following the failure to disclose a patient death, a BMO survey suggests.

The crisis over Sarepta Therapeutics' Duchenne therapy offers a valuable lesson: Listening to critics, and responding with ...

The U.S. Food and Drug Administration on Friday placed Sarepta Therapeutics’ gene therapy trials for limb girdle muscular ...

A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of ...

The third death occurred in an adult male with limb girdle muscular dystrophy who received a Sarepta AAVrh74 gene therapy product, SRP-9004, in a phase 4 clinical trial.

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

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

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

Separately, the FDA on Friday informally requested that Sarepta voluntarily pause shipments of another gene therapy, Elevidys, that is approved to treat a different type of muscular dystrophy and was ...

FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due ...

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