News

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
An unusual public feud between the Food and Drug Administration and a maker of gene therapies for rare diseases could test ...
The FDA on Tuesday rejected a skin therapy from Replimune Group, suggesting a hardened stance on drug approvals under new agency officials ...
Rocket Pharmaceuticals jumps 16.8% as FDA grants RMAT status to RP-A601 on positive phase I heart disease data.