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

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

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

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

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

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

Roche Holding AG’s gene therapy Elevidys failed to get the backing of European regulators, a blow to the Swiss drugmaker and to Sarepta Therapeutics Inc., the embattled US biotech that developed the ...

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...

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 ...

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...