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Phase II trial of acetazolamide for cerebellar syndrome in PMM2-CDG
2017-000810-44Phase 2Completed
- Sponsor
- Fundació Sant Joan de Déu
- Start
- Apr 2017
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, single-arm trial of acetazolamide in phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG) and cerebellar dysfunction, sponsored by Fundació Sant Joan de Déu. Primary endpoint: Movement disorder improvement. Started Apr 2017; status: completed.
02
Drugs
acetazolamide
03
Conditions
phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG)cerebellar dysfunction
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