New drug tested to help kids with rare genetic disorder
Disease control
Completed
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 t…
Phase: PHASE2, PHASE3 • Sponsor: Applied Therapeutics, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC