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There are no effective therapies for the vast number of RARE NEUROLOGICAL DISEASES
Neurogene is accelerating development of new genetic medicines to people with devastating neurological diseases and their families. To do this, we are working with experts across the globe to advance a broad pipeline of programs to treat the underlying cause of serious neurological disorders and thereby address the overwhelming need for new therapies.

Although the human genome was sequenced for the first time nearly 20 years ago, the true genomics revolution is taking place now, providing us with the ability to radically alter rare, genetic disorders. At Neurogene, we are working to provide medicines to improve the lives of neurologically-impaired and developmentally-delayed children and their families.

For children, our goal is simple: to enable them to have a healthier future. For their families, we want to make their lives better: when one family member is neurologically impaired, there is a heavy burden on caregivers – whether it is caring for an infant who will not live to see her third birthday, communicating with a son who doesn’t speak, or attempting to control a teenager in the throes of a self-injurious tantrum.

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