Experimental gene therapy offers hope for children with rare brain disease
NCT ID NCT04833907
First seen Nov 14, 2025 · Last updated May 14, 2026 · Updated 23 times
Summary
This study tests a one-time gene therapy, called AVASPA, for children with Canavan disease, a rare and severe brain disorder. The therapy is given directly into the brain during a surgical procedure. The goal is to restore the missing enzyme and improve brain function. The trial enrolls 24 children up to 5 years old.
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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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