Gne Myopathy
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
A completed natural history observational study of patients with GNE myopathy and related GNE disorders.
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: First-in-human Study of UX016 in GNEM [Phase 1, Phase 2]
Update on a first-in-human study of UX016 for GNE myopathy (GNEM) in Phase 1/2, currently not recruiting.
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CLINICAL_TRIALS research · 2025-06-13Clinical Trial Update: Multi-Center Study of ManNAc for GNE Myopathy [Phase 2]
A Phase 2 multi-center trial evaluating ManNAc for GNE myopathy is currently active but not recruiting.
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CLINICAL_TRIALS research · 2018-04-27Clinical Trial Update: GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
This clinical trial update describes Ultragenyx's GNE-Myopathy Disease Monitoring Program, a registry and prospective natural history study.…