Gene therapy hope for kids with rare nerve disease
NCT ID NCT04283227
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 31 times
Summary
This study tests a one-time gene therapy called OTL-200 in 6 people with late juvenile metachromatic leukodystrophy (MLD), a rare genetic disease that damages the nervous system. The treatment uses the patient's own blood stem cells, modified to produce a missing enzyme, to slow or stop disease progression. The goal is to see if it safely improves brain health and function.
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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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