Gene therapy shows promise for rare, devastating childhood disease
NCT ID NCT03392987
First seen Mar 18, 2026 · Last updated May 18, 2026 · Updated 6 times
Summary
This study tested a one-time gene therapy (OTL-200) in 10 children with early-onset metachromatic leukodystrophy (MLD), a severe genetic disorder that damages the nervous system. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme. The main goal was to see if it could preserve motor function. While not a cure, it aims to control the disease and improve quality of life.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, 20132, Italy
Conditions
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