New hope for kids with rare brain disease: drug trial targets sanfilippo
NCT ID NCT06181136
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 29 times
Summary
This study tests a new drug called DNL126 in 20 children with Sanfilippo syndrome type A, a rare genetic disease that damages the brain and body. The goal is to see if the drug is safe and can lower harmful substances linked to the disease. The study lasts about 6 months, with options to continue for up to 4 years.
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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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Baylor College of Medicine and Texas Children's Hospital
Houston, Texas, 77030, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
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University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514, United States
Conditions
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