Gene therapy breakthrough for rare childhood epilepsy?
NCT ID NCT06283212
First seen Jan 05, 2026 · Last updated May 24, 2026 · Updated 18 times
Summary
This study tests a new gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The treatment aims to reduce seizures and improve development by delivering a working gene to brain cells. The study is early-stage (Phase 1/2) and focuses on safety, but also measures how well it controls the disease.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
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Queen Elizabeth Hospital
Glasgow, G51 4TF, United Kingdom
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Sheffield Children's Hospital
Sheffield, S10 2TH, United Kingdom
Conditions
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