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2 Girls Become World s First To Get Gene Therapy For Brain Disorder

2 Girls Become World s First To Get Gene Therapy For Brain Disorder
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New Gene Therapy to Treat Genetic Brain Disorder

2 US girls become world s first to get gene therapy for genetic brain disorder

A team of US doctors have in a world first delivered a novel gene therapy to treat Rett syndrome a rare genetic brain disorder in two girls. They became the first children worldwide to receive the promising treatment as part of the first-in-human Phase I/II trial of a new investigational gene therapy, NGN-401, developed by US-based Neurogene.

2 US girls become world s first to get gene therapy for genetic brain disorder

A team of US doctors have in a world first delivered a novel gene therapy to treat Rett syndrome a rare genetic brain disorder in two girls. They became the first children worldwide to receive the promising treatment as part of the .

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