New drug shows promise for kids with rare sugar disorder
Disease control
Completed
This study tested a drug called AT-007 in 47 children with classic galactosemia, a rare genetic condition 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, and also lower a harmful…
Phase: PHASE2, PHASE3 • Sponsor: Applied Therapeutics, Inc. • Aim: Disease control
Last updated Apr 29, 2026 15:05 UTC