Gene therapy survivors tracked for years in rare brain disease study
NCT ID NCT04273243
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 26 times
Summary
This study follows 10 people with CLN6 Batten disease who previously received a one-time gene therapy injection into the spine. Researchers are checking for long-term side effects and using a standard scale to measure how the disease progresses over time. The goal is to understand the lasting safety and effects of the treatment.
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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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