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 ...

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 ...

Sarepta Therapeutics Inc. shares rose sharply early Tuesday after US regulators reversed course and recommended that patients ...

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

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

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

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...