CLASSIC GALACTOSEMIA
Clinical trials for CLASSIC GALACTOSEMIA explained in plain language.
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New hope for kids with rare sugar disorder: drug shows promise in trial
Disease control CompletedThis study tested an experimental drug called AT-007 in 47 children with classic galactosemia, a rare genetic disorder 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, while also …
Matched conditions: CLASSIC GALACTOSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Applied Therapeutics, Inc. • Aim: Disease control
Last updated May 22, 2026 13:51 UTC
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Babble boot camp aims to prevent speech delays in rare disease
Prevention CompletedThis study tested a program called Babble Boot Camp for babies with classic galactosemia, a rare condition that often causes speech and language delays. The program involved weekly online coaching for parents from a speech therapist, starting when babies were 2 months old. The go…
Matched conditions: CLASSIC GALACTOSEMIA
Phase: EARLY_PHASE1 • Sponsor: Arizona State University • Aim: Prevention
Last updated May 22, 2026 13:55 UTC