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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

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Rare Disease Research Center

    Atlanta, Georgia, 30318, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

Conditions

Explore the condition pages connected to this study.