Gene therapy for rare childhood brain disease: tracking kids for years after treatment
NCT ID NCT04360265
Summary
This study is tracking children with Sanfilippo Syndrome A (MPS IIIA) who received an experimental gene therapy called UX111 in earlier trials. The goal is to monitor their long-term safety and see how well the treatment continues to work over many years. Researchers will check for side effects and measure brain development and disease markers. No new treatment is given in this study—it's purely for observation and data collection.
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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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