New hope for kids with rare sugar disorder: drug shows promise in trial
NCT ID NCT04902781
First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 22 times
Summary
This study tested an experimental drug called AT-007 in 47 children with classic galactosemia, a rare genetic disorder that makes it hard to break down a sugar called galactose. The goal was to see if the drug could improve behavior, daily living skills, and language, while also reducing harmful substances in the blood. The trial is complete, but results are not yet public.
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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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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Rare Disease Research Center
Atlanta, Georgia, 30318, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
Conditions
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