Gene therapy offers hope for kids with rare brain disease
NCT ID NCT02716246
First seen Nov 06, 2025 · Last updated May 22, 2026 · Updated 30 times
Summary
This study tests a gene therapy called UX111 for children with Sanfilippo syndrome type A (MPS IIIA), a rare genetic disease that damages the brain. The treatment delivers a working gene to help the body break down harmful substances. The goal is to slow or stop the disease, but children will still need ongoing care and monitoring.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of Pittsburgh
COMPLETEDPittsburgh, Pennsylvania, 15224, United States
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Hospital Clínico Universitario de Santiago
RECRUITINGSantiago de Compostela, 15706, Spain
Contact Email: •••••@•••••
Contact Email: •••••@•••••
Contact
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Nationwide Children's Hospital
COMPLETEDColumbus, Ohio, 43205, United States
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Vall d'Hebron Barcelona Hospital Campus
RECRUITINGBarcelona, 08035, Spain
Contact Email: •••••@•••••
Contact
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Women's and Children's Hospital
COMPLETEDNorth Adelaide, South Australia, 5006, Australia
Conditions
Explore the condition pages connected to this study.