News

FDA Says Sarepta Can Resume Gene Therapy in Some Patients
In a major win for Sarepta Therapeutics Inc., US regulators are recommending that patients who can walk be allowed to take ...
Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
Sarepta fails to win EU backing for muscle disorder gene therapy
Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...
MIT Technology Review6d
The deadly saga of the controversial gene therapy Elevidys
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
FDA opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
FDA Probes Death of Patient on Sarepta's Elevidys, Partner Roche Says Death Unrelated to Therapy
(Reuters) -The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who ...
Medscape5d
EMA Says No to Duchenne Gene Therapy Elevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
Chemistry World1d
Sarepta gene therapy deaths highlight tragic rare disease dilemma
Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...
Fierce Pharma4h
Vinay Prasad's exit signals FDA's probable slip toward more permissive regulation of cell and gene therapy: analyst
But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...
The American Journal of Managed Care4h
FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients
Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...
Fierce Pharma1d
Vinay Prasad departs FDA amid conservative criticism, controversy over Sarepta gene therapy
Vinay Prasad, M.D., has left the FDA less than three months into the role as director of the Center for Biologics Evaluation ...
GlobalData on MSN23h
Prasad’s FDA exit may accelerate cell and gene therapy approvals
Vinay Prasad, head of the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA), ...