Limb-girdle muscular dystrophy (LGMD) refers to a group of inherited muscle disorders characterized by progressive weakness ...

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

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

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

The number of biopharma professionals let go has increased year over year for three straight months. In July, nearly 8,000 ...

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

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

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

President Trump's executive order to "identify and take appropriate action to correct past misconduct by the Federal Government related to censorship of protected speech" is good enough, DOJ tells ...

CAMBRIDGE, Mass., Aug. 04, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced the appointment of ...

Vinay Prasad, a professor who until earlier this year headed a cancer-drugs and health-policy lab at UC San Francisco, ...

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