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

US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

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

SRPT rallies after Q2 earnings and revenue beat expectations as Elevidys deliveries resume following FDA clearance.

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 ...

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

This is the web edition of D.C. Diagnosis, STAT's twice-weekly newsletter about the politics and policy of health and ...

The FDA had halted use in older adults while it investigated reports of side effects. The vaccine’s label now carries new ...