Shortly after the U.S. FDA announced an investigation into the death of a patient who had received Elevidys, a gene therapy ...

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 ...

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 ...

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 ...

Elevidys, which is also facing regulatory hurdles in the US, was developed by Sarepta Therapeutics but is sold by Roche outside of the US.

This week's episode covers news about Sarepta, babies born following mitochondrial transfer, and AI identifying therapy targets.