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

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

Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...

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

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

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.

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy ...