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 ...

Rare disease and gene therapy stocks, battered in recent months by clinical and commercial setbacks, will likely benefit from ...

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

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 ...

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 ...

The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.