The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

Shares of Sarepta Therapeutics Inc (NASDAQ:SRPT) plunged 10% after the company disclosed that the U.S. Food and Drug ...

Sarepta Therapeutics has paused U.S. shipments of its gene therapy Elevidys after two teenagers with Duchenne muscular ...

The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...

The FDA asked Sarepta Therapeutics to halt shipments of its Elevidys gene therapy after a patient's death linked to a similar treatment. Sarepta will continue shipments for ambulatory patients but ...

Edgewise Therapeutics Inc. (NASDAQ:EWTX) saw its shares slide by 7.2% after the U.S. Food and Drug Administration (FDA) concluded that current data from the company’s CANYON trial does not meet ...

WINDHAM, N.H. — Despite being diagnosed with autism and Duchenne muscular dystrophy, 8-year-old Connor Mullaly is always sporting a smile.