Gene therapy injection aims to control rare brain disease in kids
NCT ID NCT07603050
First seen May 25, 2026 · Last updated May 25, 2026
Summary
This early-stage study tests a new gene therapy called VGN-R08b, given as a single injection into the brain fluid, for children aged 2 to 17 with Type III Gaucher's disease. The goal is to see if it is safe and can improve enzyme activity related to the disease. Only 12 participants will be enrolled, and the study is not yet recruiting.
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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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