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

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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the ...

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

The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...

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

A similar but perhaps less dramatic story played out earlier in July, when Capricor Therapeutics, Inc. received a CRL for its ...

A 15-year-old boy in New Market with a neuromuscular disorder received a wheelchair accessible van last week through the support of family, community and a nonprofit dedicated to assisting those with ...

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 ...

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

Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock declined 13% Friday after European regulators recommended against approving Elevidys, the company’s gene therapy for Duchenne muscular dystrophy.

The Swiss pharmaceutical group said the negative opinion from the European Medicines Agency was based on data that included a phase 3 study in which Elevidys didn't meet its primary endpoint after one ...