Brain gene injection shows promise for rare childhood disease
NCT ID NCT01801709
First seen Nov 18, 2025 · Last updated May 26, 2026 · Updated 20 times
Summary
This study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare and severe brain disease. Five children aged 6 months to 5 years received a single injection of a harmless virus carrying a healthy gene directly into their brains. The goal was to see if it was safe and could slow or stop the disease from getting worse.
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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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Bicêtre Hospital - Paris Sud
Le Kremlin-Bicêtre, France
Conditions
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