Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...

Good morning! Today, we get deeper into the ousting of Nicole Verdun at the FDA, see promise from Vertex’s cell therapy for ...

Capricor stock drops as FDA cancels advisory meeting on Duchenne therapy; leadership changes and regulatory tensions fuel uncertainty.

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.

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Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and ...

Nicole Verdun, MD, super office director of the FDA’s Office of Therapeutic Products (OTP) and her deputy Rachael Anatol, PhD ...

He was diagnosed with Duchenne muscular dystrophy at age 5.Family remembers the day everything changed"I can remember the day ...

A North Texas family is sharing a son's journey with Duchenne muscular dystrophy, a rare and progressive muscle-wasting ...

Sarepta shares face pressure after a second Elevidys-linked death and analyst downgrade over safety concerns and lowered revenue prospects.

The Kansas City Chiefs welcomed some special guests to their mandatory minicamp on Tuesday, June 17, and Patrick Mahomes and ...