The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.

Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the FDA.

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...

Sarepta Therapeutics announces voluntary pause all shipments of Elevidys for Duchenne muscular dystrophy in US: Cambridge, Massachusetts Wednesday, July 23, 2025, 12:00 Hrs [IST] ...

The Food and Drug Administration named a longtime pharmaceutical executive to run the agency’s drug program. Dr.

Most Americans encounter the Federal Trade Commission only if they’ve been scammed: It handles identity theft, fraud, and ...

CMS’s Innovation Center plans to expand its workforce as it prepares to launch new health care payment models.

Medicaid and ACA, the homeless crisis, children's health, 'black box' drug warnings, nutrition and SNAP, maternity care, and more.

CAMBRIDGE, Mass., July 21, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced the appointment of ...

A breakthrough in trade talks with the U.S. might win Ishiba a reprieve, but so far there's been scant sign of progress in negotiating away the threat of higher tariffs on Japan 's exports to the U.S.

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA ...