duchenne muscular dystrophy gene therapy

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Roche pauses Sarepta gene therapy shipments outside U.S.

Roche halts some shipments of Sarepta’s Duchenne gene therapy outside U.S.

Roche is following its partner Sarepta Therapeutics and stopping shipments of the Duchenne muscular dystrophy gene therapy Elevidys in some countries, amid rising safety concerns surrounding the treat...

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Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

Becker's Hospital Review · 5h

Roche pauses gene therapy shipments

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InnovationRx: Sarepta Blinks In Showdown With FDA

Last week, in a remarkable showdown, Sarepta Therapeutics refused to stop distribution of Elevidys, its therapy for the muscle-wasting disease Duchenne muscular dystrophy as the FDA had requested.

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Sarepta falls after gene therapy maker pauses Elevidys shipments in US

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Sarepta Faces Fresh Setback. Why Now’s Not the Time to Buy the Stock.

STAT · 1d

Sarepta Therapeutics’ Duchenne therapy faces ‘arduous’ path back to market, senior FDA official says

But it would be difficult for Sarepta to prove that any new safety protocols could eliminate the risk of liver injuries — which have been tied to the deaths of two patients — the official added.

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Sarepta pares gains as report suggests an ‘arduous’ path for Duchenne drug

STAT · 1d

In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

The BMJ17h

Duchenne gene therapy halted after three deaths and company u turn

The biotechnology company Sarepta Therapeutics has decided to suspend entirely one of its treatments for Duchenne muscular dystrophy (DMD) just days after it refused a request from the US Food and Drug Administration (FDA) to pull the drug from the market.

3don 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 gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.

Zacks Investment Research on MSN2d

SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its experimental gene therapies. The company confirmed that a 51-year-old, non-ambulant (unable to walk) limb-girdle muscular dystrophy (LGMD) patient died due to acute liver failure (ALF).

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 ...

Fierce Biotech2d

Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle muscular dystrophy (LGMD) trials, while also revoking the biopharma’s gene therapy platform technology designation.

STAT5dOpinion

We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we never asked to join — said goodbye to their sons, the babies they once held in their arms, whose dreams they held in their hearts until Duchenne robbed them of working muscles or a healthy future.

Muscular Dystrophy News6d

Dosing begins in gene therapy trial for DMD-linked heart disease

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated with Duchenne MD.