Gene therapy shows promise for rare, devastating brain disease in kids
NCT ID NCT03392987
First seen Mar 18, 2026 · Last updated May 15, 2026 · Updated 5 times
Summary
This study tested a gene therapy called OTL-200 in 10 children with early-onset metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme, and aims to slow or stop disease progression. Researchers measured motor function to see if the therapy helped maintain movement abilities.
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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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Contacts and locations
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Locations
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Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, 20132, Italy
Conditions
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