The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

(Reuters) -The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who ...

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.

BASEL, Switzerland I July 25, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the ...

Nucleic acid therapies offer targeted treatment for genetic diseases, but delivery remains a key hurdle. Explore how advances ...

The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta ...

US regulators are investigating the death of an 8-year-old boy in Brazil who received Sarepta Therapeutics Inc.’s Elevidys.

The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics ( NASDAQ: SRPT) gene therapy for Duchenne ...

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the ...

After a tough few weeks for Sarepta Therapeutics Inc., the EMA dealt another blow on July 25, announcing it will not be ...