Phosphomannomutase Deficiency
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CLINICAL_TRIALS trial_result · 2025-12-17Clinical Trial Update: 24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG [Phase 2]
A Phase 2 24-week trial evaluating GLM101 in PMM2-CDG has completed, reporting on pharmacodynamics, safety, tolerability, and pharmacokineti…
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CLINICAL_TRIALS research · 2025-11-18Clinical Trial Update: Natural History Study Protocol in PMM2-CDG (CDG-Ia)
This document describes a natural history study protocol for PMM2-CDG (phosphomannomutase 2 deficiency) sponsored by Glycomine, Inc., with s…
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CLINICAL_TRIALS trial_result · 2025-09-25Clinical Trial Update: Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG [Phase 3]
A Phase 3 trial evaluating oral epalrestat in pediatric subjects with PMM2-CDG has been terminated.