Gene therapy offers hope for kids with fatal brain disorder
NCT ID NCT02725580
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 28 times
Summary
This study tested a one-time gene therapy for children with a rare, severe brain disease called variant late infantile Batten disease. The treatment delivers a working copy of the CLN6 gene directly into the spinal fluid to help slow the loss of motor and language skills. Thirteen children with mild to moderate symptoms took part. The main goals were to check safety and see if the therapy could slow the disease's progression.
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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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Nationwide Children's Hosptial
Columbus, Ohio, 43205, United States
Conditions
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