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

After Loomer targeted surgeon general nominee and Kennedy ally Casey Means, Means’s brother, Calley, suggested that Loomer’s ...

The cost of high-end, breakthrough medications traumatizes patients and their families. According to USAfacts, “national ...

We expect Sarepta Therapeutics SRPT to report second-quarter 2025 earnings on Aug. 6, after market close. The company’s ...

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 ...

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

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

Vinay Prasad's interim replacement has concerns about same drug that drove him out. NIH director says employee critics ...

The FDA has denied that it plans to combine the Center for Drug Evaluation and Research and Center for Biologics Evaluation ...

Two major health agencies are facing leadership reshuffles, with the resignations of the FDA’s Vinay Prasad and NICE’s Dr Sam Roberts.

PTC Therapeutics faces pivotal FDA decisions for approvals that could potentially transform their long-term revenues. Click ...

The U.S. Food and Drug Administration's (FDA) top vaccine official is stepping down after just three months in a role that ...