New drug trial aims to ease debilitating effects of rare genetic disorder in kids
NCT ID NCT04902781
Summary
This study tested whether a drug called AT-007 could improve daily life for children with classic galactosemia, a rare genetic disorder. Researchers measured changes in behavior, language skills, thinking, and motor abilities in kids aged 2 to 17. The goal was to see if long-term treatment could help control the disease's neurological effects.
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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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