Stem cell gene therapy offers hope for children with fatal brain disease
NCT ID NCT01560182
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 29 times
Summary
This study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare and fatal genetic disease that damages the brain and nerves. Doctors took stem cells from each child, added a working copy of the faulty gene, and put the cells back after mild chemotherapy. The goal was to slow or stop the disease. The trial included 20 children who were either not yet showing symptoms or had early signs of the disease.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for METACHROMATIC LEUKODYSTROPHY are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, 20132, Italy
Conditions
Explore the condition pages connected to this study.