One-Time gene therapy tested to halt devastating childhood brain disease
NCT ID NCT04283227
Summary
This study is testing a single infusion of an experimental gene therapy called OTL-200 in up to 6 children with late juvenile metachromatic leukodystrophy (MLD), a rare and severe genetic brain disease. The therapy aims to correct the underlying genetic cause by giving the patient's own modified blood stem cells back to them. The main goals are to see if the treatment is safe and if it can slow or stop the progression of the disease by measuring changes in brain chemistry and function.
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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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