gene therapy duchenne muscular dystrophy

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Duchenne, European Medicines Agency and gene therapy

Sarepta woes mount as Duchenne gene therapy knocked back in Europe

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could further jeopardize Sarepta’s financial future.

STAT · 10h

For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak

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

The Manila Times · 4h

Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU

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 dialogue with the EMA to explore a potential path forward to make Elevidys available to individuals living with DMD in the EURoche believes the benefit-risk remains positive in the ambulatory Duchenne populationElevidys is the first and only disease-modifying gene therapy for DMD Basel,

STAT4h

Europe moves to reject embattled Duchenne muscular dystrophy gene therapy

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

Medscape5m

EMA Says No to Duchenne Gene TherapyElevidys

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

MIT Technology Review6h

The deadly saga of the controversial gene therapy Elevidys

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

17mon MSN

Sarepta fails to win EU backing for muscle disorder gene therapy

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

InvestorsHub on MSN1h

Sarepta shares drop after EU regulators reject Elevidys gene therapy

Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) plunged 13% on Friday after a major regulatory blow in Europe. The European ...

Fierce Pharma1h

Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe

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

USA Today2y

Duchenne muscular dystrophy treatments, gene therapy spark hope - USA TODAY

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 ...

4don MSN

FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths

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

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