Congenital Disorder of Glycosylation Type Ia
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CLINICAL_TRIALS trial_result · 2025-03-20Clinical Trial Update: Acetazolamide Efficacy in Ataxia in PMM2-CDG [Phase 2, Phase 3]
A Phase 2/3 clinical trial evaluating acetazolamide for ataxia in PMM2-CDG/CDG-Ia was terminated, with results posted.