CLASSIC GALACTOSEMIA
Clinical trials for CLASSIC GALACTOSEMIA explained in plain language.
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New drug tested to help kids with rare genetic disorder
Disease control CompletedThis 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…
Matched conditions: CLASSIC GALACTOSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Applied Therapeutics, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC
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Can baby babble training stop speech disorders before they start?
Prevention CompletedThis study tested whether early speech and language coaching could prevent communication problems in babies with classic galactosemia, a genetic condition that puts them at high risk for speech disorders. Researchers worked with 285 families, providing weekly online training to p…
Matched conditions: CLASSIC GALACTOSEMIA
Phase: EARLY_PHASE1 • Sponsor: Arizona State University • Aim: Prevention
Last updated Mar 27, 2026 12:39 UTC