Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

Vinay Prasad, M.D., has left the FDA less than three months into the role as director of the Center for Biologics Evaluation ...

From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to ...

SRPT's Q2 report, set to arrive Aug. 6, is likely to have Elevidys updates, restructuring plans and sales outlook in sharp investor focus.

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

Vinay Prasad exits FDA's biologics division following pressure over Sarepta's Elevidys approval and regulatory controversies.

Extend transfection complex formation time by up to 3 hours, reduce complex volume by >50% and maintain high titers and full ...

Vinay Prasad, head of the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA), ...