Gene therapy trial aims to tame severe seizures in kids with dravet syndrome
NCT ID NCT06283212
First seen Jan 05, 2026 · Last updated May 14, 2026 · Updated 17 times
Summary
This study tests a gene therapy called ETX101 in 5 children aged 6 months to under 4 years with Dravet syndrome, a severe epilepsy. The treatment uses a harmless virus to deliver a working gene to brain cells, aiming to reduce seizures and improve development. The main goals are to check safety and see if seizures and daily living skills get better over a year.
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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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Contacts and locations
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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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