gene therapy duchenne muscular dystrophy

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Elevidys, Gene Therapy and Duchenne muscular dystrophy

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.

Medscape · 1h

EMA Says No to Duchenne Gene Therapy Elevidys

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

EconoTimes · 11h

FDA Probes Death of 8-Year-Old Linked to Sarepta’s Elevidys Gene Therapy

The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta Therapeutics (NASDAQ: SRPT) gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD).

1hon MSN

Two boys died after a gene therapy. This family won’t give up hope.

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.

STAT1d

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 ...

MIT Technology Review1d

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.

14h

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 ...

5don 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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