The family of a seven-year-old boy diagnosed with a life-changing condition are raising funds to build a specially-adapted ...

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

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 ...

Sarepta and Roche face a regulatory setback as the EMA's CHMP issues a negative opinion on Elevidys for Duchenne muscular ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

This week's episode covers news about Sarepta, babies born following mitochondrial transfer, and AI identifying therapy ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

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

Sarepta Therapeutics (NASDAQ:SRPT) faces an “arduous and treacherous path” to bring its gene therapy Elevidys back to market, ...

There were warnings to the FDA about Sarepta Therapeutics Inc. before US regulators asked the company to halt shipments of its gene therapy.