Brain gene therapy offers hope for rare childhood disease
NCT ID NCT04833907
First seen Nov 14, 2025 · Last updated May 23, 2026 · Updated 27 times
Summary
This study tests a one-time gene therapy, AVASPA, given directly to the brain in children with Canavan disease, a severe genetic disorder that damages white matter and shortens life. The therapy aims to restore a missing enzyme to help the brain make myelin and reduce harmful buildup. Up to 24 children, from newborns to age 5, will be enrolled to check safety and measure changes in brain structure and function.
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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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Dayton Children's Hospital
Dayton, Ohio, 45404, United States
Conditions
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