[HTML][HTML] OP005: AT-007 significantly reduces toxic galactitol in ACTION-galactosemia kids-the 1st therapeutic interventional clinical trial in children with classic …
E Bailey, S Wang, L Saltonstall, R Perfetti… - Genetics in …, 2022 - gimjournal.org
Methods ACTION-Galactosemia Kids is a sequential, two-part, randomized double-blind,
placebo-controlled study evaluating the clinical benefit, safety, pharmacokinetics and
pharmacodynamics of AT-007 in pediatric patients with CG. Patients were randomized 2: 1
to AT-007 or placebo. Disease demographics, genetic variant, and patient history were
assessed at baseline. Additionally, functional tests of speech, motor function, cognition and
behavior were performed at baseline for all patients enrolled in the clinical study. The …
placebo-controlled study evaluating the clinical benefit, safety, pharmacokinetics and
pharmacodynamics of AT-007 in pediatric patients with CG. Patients were randomized 2: 1
to AT-007 or placebo. Disease demographics, genetic variant, and patient history were
assessed at baseline. Additionally, functional tests of speech, motor function, cognition and
behavior were performed at baseline for all patients enrolled in the clinical study. The …