Gene therapy injection shows promise for kids with rare movement disorder
NCT ID NCT06432140
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial tests a gene therapy called VGN-R09b for children with severe AADC deficiency, a rare genetic disorder that affects movement and brain function. The therapy is injected directly into the brain. The study aims to see if it is safe and helps children achieve motor milestones like head control and walking. Only 13 children aged 18 months to 8 years are enrolled.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
VGN-R09b (a gene therapy injected into the brain)
What this could lead to
If successful, this could provide a treatment that helps children with AADC deficiency gain motor skills like head control and walking.
What could go wrong
This is an early-phase trial with only 13 participants, so results may not apply to all. Risks include side effects from the injection or the therapy not working as hoped.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Shanghai
Shanghai, Shanghai Municipality, 200120, China