Gene therapy for rare brain disease: Long-Term safety check begins
NCT ID NCT04360265
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 27 times
Summary
This study follows 41 children with Sanfilippo A (MPS IIIA) who previously received the gene therapy UX111 in an earlier trial. Researchers will monitor safety and measure how well the treatment controls the disease over time, including thinking skills and chemical markers in spinal fluid. The goal is to see if the therapy remains safe and effective in the long run.
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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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Hospital Clínico Universitario de Santiago
Santiago de Compostela, Spain
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Vall d'Hebron Barcelona Campus
Barcelona, 08035, Spain
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Women's and Children's Hospital
North Adelaide, South Australia, Australia
Conditions
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