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Genetic Therapy for Dravet Syndrome: 2022 State of the Field

When the Dravet Syndrome Foundation was established in 2009, there were no specific treatments for patients with Dravet syndrome and limited guidelines on best treatment approaches. Despite the known genetic cause for the majority of cases, mutations in SCN1A, the idea of a genetic-based therapy seemed a distant possibility at that time. Things have changed quite a bit in the last 13 years; there are now three FDA-approved medications with specific indications for Dravet syndrome, and several genetic-based therapies in varying stages of development, including one already in human clinical trials. DSF continues to fund, advocate, and support the development of new approaches to more fully treat Dravet syndrome. With the ever-changing field, DSF also strives to keep the community up-to-date. To that effort, please read our most recent update to our Gene Therapy State-of-the-Field to learn more about the genetic-based therapies that are in development for Dravet syndrome.

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