gene therapy duchenne muscular dystrophy

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Sarepta, Duchenne and Gene Therapy

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

Since then, regulators’ call for Sarepta Therapeutics to halt shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, and the company’s subsequent decision to do so, have stoked conf...

· 4d · on MSN

Sarepta’s Risky Gamble: Gene Therapy Stays on Market Despite FDA Request

· 2d

Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

· 1d

InnovationRx: Sarepta Blinks In Showdown With FDA

The latest twist in the saga followed a decision by Children’s Hospital Los Angeles to halt use of the drug while it was facing regulatory uncertainty.

· 2d

Sarepta falls after gene therapy maker pauses Elevidys shipments in US

· 2d

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

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 MSN3d

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).

Medindia12mon

Gene Therapy Offers New Hope for Duchenne Muscular Dystrophy ... - Medindia

Gene therapy approach developed offers new hope for those with Duchenne muscular dystrophy by restoring the full-length dystrophin protein. Systemic delivery of full-length dystrophin in Duchenne ...

Science News2y

The first gene therapy for muscular dystrophy has been approved for ...

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

News Medical1y

Breakthrough gene therapy offers hope for Duchenne muscular dystrophy

In the case of Duchenne muscular dystrophy – the topic of this study – patients, all males because the gene is on the X chromosome, begin exhibiting symptoms around age four and usually die ...