The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...

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

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

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a ...