- CDG Type(s): PMM2-CDG
- Study Type: Clinical Trial, Phase II/III
- Location: United States, Multi-Centre
- Status: Terminated
Mayo Clinic, in collaboration with Seattle Children’s Hospital and Children’s Hospital of Philadelphia, is conducting an interventional, randomized, double-blind, placebo-controlled study on PMM2-CDG. The purpose of this study is to assess efficacy of acetazolamide treatment of improving ataxia in PMM2-CDG patients, while monitoring adverse reactions, effects on biomarkers and characteristics of patients who do not respond to acetazolamide treatment.
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