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

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

· 17h

Sarepta woes mount as Duchenne gene therapy knocked back in Europe

· 9h · on MSN

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

STAT20h

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 Review22h

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.

8hon MSN

FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy

The patient with Duchenne muscular dystrophy, who died in Brazil, was treated with Elevidys but was not a participant in a ...

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 ...

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