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MedPage Today2d
Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
The BMJ7h
Duchenne gene therapy halted after three deaths and company u turn
The biotechnology company Sarepta Therapeutics has decided to suspend entirely one of its treatments for Duchenne muscular dystrophy (DMD) just days after it refused a request from the US Food and ...
FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
MedPage Today on MSN6d
Duchenne Gene Therapy Will Undergo Changes After Patient Deaths
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
Sarepta Therapeutics crisis is huge blow to Duchenne families, company
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
USA Today2y
Duchenne muscular dystrophy treatments, gene therapy spark hope - USA TODAY
Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...