gene therapy duchenne muscular dystrophy

News

Duchenne, Sarepta and gene therapy

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.

Yahoo Finance · 10h

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.

The Boston Globe · 3d

Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

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

Deadly saga of the controversial gene therapy Elevidys

MIT Technology Review · 15h

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.

U.S. News & World Report · 1h

US FDA Investigates Third Death After Treatment With Sarepta's Gene Therapy Elevidys

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received Sarepta Therapeutics' muscular disorder gene therapy Elevidys. The death occurred on June 7,

Bloomberg on MSN · 12h

Sarepta-Roche Gene Therapy Fails to Get EU Regulator’s Nod

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 treatment.

STAT12h

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 ...

1hon MSN

FDA Investigates Death of Boy Who Got Sarepta’s Gene Therapy

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

13h

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 ...

Health Beat: Duchenne muscular dystrophy

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

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 ...

Medscape8h

EMA Says No to Duchenne Gene TherapyElevidys

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

Labroots2y

First Gene Therapy to Treat Duchenne Muscular Dystrophy is Approved

The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene ...

Results that may be inaccessible to you are currently showing.

Hide inaccessible results