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

2 US girls become world's first to get gene therapy for 'Rett syndrome' in girls

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. Dec 4, 2023
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