Clinical Trial Update: Acetazolamide Efficacy in Ataxia in PMM2-CDG [Phase 2, Phase 3]

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Summary

A Phase 2/3 clinical trial evaluating acetazolamide for ataxia in PMM2-CDG/CDG-Ia was terminated, with results posted.

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Diseases / indications

• Congenital Disorder of Glycosylation Type Ia
• Pmm2-cdg

Drugs mentioned

• ACETAZOLAMIDE (acetazolamide)

Snippet

Sponsor: Mayo Clinic | Conditions: Pmm2-CDG, CDG1A | Phase 2, Phase 3 | Status: TERMINATED | Results posted: 2025-03-20