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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 ...
Solid Biosciences said federal regulators have granted its fast-track designation for the company's investigational gene therapy for the treatment of a heart rhythm condition.
The FDA approves the first of two gene therapies this month to treat sickle cell disease. Though expensive and difficult, it promises a better life. Hotspots ranked Start the day smarter ☀️ ...
Rocket Pharmaceuticals jumps 16.8% as FDA grants RMAT status to RP-A601 on positive phase I heart disease data.
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