The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

The patient with Duchenne muscular dystrophy, who died in Brazil, was treated with Elevidys but was not a participant in a ...

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

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

US regulators said they are investigating the death of an 8-year-old boy who received a gene therapy from Sarepta ...

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

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

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 ...

Sarepta Therapeutics Inc. shares plunged after European regulators rejected its gene therapy Elevidys, intensifying scrutiny on the drugmaker after it was pressured to halt shipments of its treatment ...

Friday said that the European Medicines Agency has issued a negative opinion on the conditional marketing authorization for Elevidys, intended for ambulatory patients aged three to seven with Duchenne ...