Gene therapy offers hope for children with rare movement disorder
NCT ID NCT06432140
First seen Apr 06, 2026 · Last updated May 23, 2026 · Updated 7 times
Summary
This trial tests a gene therapy called VGN-R09b in 13 children with severe AADC deficiency, a rare genetic disorder that causes severe movement problems. The therapy is injected directly into a brain region to help restore missing enzyme activity. The study checks safety and whether children can achieve motor milestones like head control and walking.
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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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Contacts and locations
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Locations
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Shanghai
Shanghai, Shanghai Municipality, 200120, China
Conditions
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