Records containing: Hereditary Inclusion Body Myopathy
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CLINICAL_TRIALS study record update · 2020-10-06International GNE Myopathy Patient Registry (GNE001)
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CLINICAL_TRIALS study record update · 2019-12-04Pilot Study of the Use of Intravenous Immune Globulin in Hereditary Inclusion Body Myopathy
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CLINICAL_TRIALS study record update · 2019-02-19A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment
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CLINICAL_TRIALS study record update · 2019-02-19Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
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CLINICAL_TRIALS study record update · 2018-07-09A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
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CLINICAL_TRIALS study record update · 2018-04-27GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
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CLINICAL_TRIALS study record update · 2016-06-16A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
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CLINICAL_TRIALS study record update · 2011-09-08Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)