Gene therapy injection shows promise for rare childhood movement disorder
NCT ID NCT06432140
First seen Apr 06, 2026 · Last updated May 03, 2026 · Updated 5 times
Summary
This trial tests a gene therapy called VGN-R09b for children aged 1.5 to 8 years with severe AADC deficiency, a rare genetic disorder that severely limits movement. The treatment is injected directly into a brain region called the putamen. The study aims to see if it is safe and can help children achieve motor milestones like head control and walking. 13 children are enrolled.
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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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Shanghai
Shanghai, Shanghai Municipality, 200120, China
Conditions
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