Gene therapy injection aims to control rare brain disease in children
NCT ID NCT07603050
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a new gene therapy called VGN-R08b for children with type III Gaucher disease, a rare genetic disorder affecting the brain and body. The treatment is given as a single injection into the brain's fluid spaces. The study will monitor safety and measure changes in disease markers. Only 12 participants aged 2 to 17 will be enrolled.
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Conditions
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