Distal Myopathies - 11 Studies Found Active, not recruiting : Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) : Hereditary Inclusion Body Myopathy Distal Myopathy With Rimmed : 2015-02-27 : Drug: Sialic Acid Tablets Othe Completed : Pharmacokinetic Study on N-acetylneuraminic Acid : Nonaka Myopathy Hereditary Inclusion Body Myopathy : 2010-11-08 : Drug: NPC-09 NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride) Enrolling by invitation : Study to Evaluate the Safety and Efficacy of Ace-ER Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy : Hereditary Inclusion Body Myopathy Distal Myopathy With Rimmed : 2016-04-04 : Drug: Aceneuramic Acid Extended-Release Tablets Other Names: UX001 Completed : 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) : Hereditary Inclusion Body Myopathy GNE Myopathy Nonaka Dis : 2013-02-04 Completed : A 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 : Hereditary Inclusion Body Myopathy Distal Myopathy With Rimmed : 2016-03-24 : Drug: Aceneuramic Acid Extended-Release Tablets Other Names: Ace-ER Completed : A Phase 2 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 : GNE Myopathy Hereditary Inclusion Body Myopathy : 2012-01-17 : Drug: Sialic Acid Extended Release (SA-ER) Completed : Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) : Hereditary Inclusion Body Myopathy (HIBM) GNE Myopathy : 2012-07-03 : Drug: ManNAc Single dose Recruiting : A Natural History Study of Patients With GNE Myopathy : Hereditary Inclusion Body Myopathy : 2011-08-13 Completed : Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM) : Hereditary Inclusion Body Myopathy (HIBM) : 2011-05-20 : Drug: Sialic Acid Extended Release (SA-ER) Tablets Completed : Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders : Amyotrophic Lateral Sclerosis Frontotemporal Dementia PLS< : 2011-10-21 Next >>>
