New drug tested to help kids with rare genetic disorder
NCT ID NCT04902781
Summary
This study tested whether a drug called AT-007 could help children with classic galactosemia, a rare genetic disorder. Researchers measured if the drug improved daily skills, behavior, language, and thinking compared to a placebo. The trial involved 47 children and teens aged 2 to 17 to find the best dose and see if long-term treatment was beneficial.
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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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