Gene therapy for fatal brain disease: Long-Term safety check
NCT ID NCT04273243
First seen Nov 01, 2025 · Last updated Apr 23, 2026 · Updated 24 times
Summary
This study follows 10 children with CLN6 Batten disease, a rare and fatal brain disorder, who previously received a single dose of gene therapy. Researchers are checking for long-term side effects and measuring how the disease progresses over time. The goal is to understand if the treatment remains safe and helps slow 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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Locations
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Nationwide Children's Hosptial
Columbus, Ohio, 43205, United States
Conditions
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