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Second patient death from liver failure after Sarepta's Elevidys gene therapy triggers FDA investigation. Stock crashes 41% ...
In the case of Duchenne muscular dystrophy – the topic of this study – patients, all males because the gene is on the X chromosome, begin exhibiting symptoms around age four and usually die ...
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying the agency was investigating the three deaths due to acute liver failure ...
Gene therapy approach developed offers new hope for those with Duchenne muscular dystrophy by restoring the full-length dystrophin protein. Systemic delivery of full-length dystrophin in ...
Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its ...
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...