The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...

The FDA’s top vaccine and gene therapy official resigned amid heightened scrutiny over recent drug approval decisions and ...

Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its ...

Sarepta Therapeutics said it will resume shipments of its gene therapy Elevidys® (delandistrogene moxeparvovec-rokl) “imminently” to ambulant patients with Duchenne muscular dystrophy (DMD), ending a ...

Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the FDA.

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

The Food and Drug Administration is investigating Sarepta’s gene therapy products following reports of a third death from acute liver failure.

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

Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its gene therapy, Elevidys. Read more here.

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