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Distal Myopathies - 11 Studies Found

Status Study
Active, not recruiting Study Name: Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Condition:
  • Hereditary Inclusion Body Myopathy
  • Distal Myopathy With Rimmed
    Date: 2015-02-27
    Interventions:
    • Drug: Sialic Acid Tablets Othe
Completed Study Name: Pharmacokinetic Study on N-acetylneuraminic Acid
Condition:
  • Nonaka Myopathy
  • Hereditary Inclusion Body Myopathy

Date: 2010-11-08
Interventions: Drug: NPC-09 NPC-09 tablet NPC-09 contains 50mg or 100mg N-acetylneuraminic acid (anhydride)
Enrolling by invitation Study Name: Study to Evaluate the Safety and Efficacy of Ace-ER Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
Condition:
  • Hereditary Inclusion Body Myopathy
  • Distal Myopathy With Rimmed
    Date: 2016-04-04
    Interventions: Drug: Aceneuramic Acid Extended-Release Tablets Other Names:
    • UX001
Recruiting Study Name: 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)
Condition:
  • Hereditary Inclusion Body Myopathy
  • GNE Myopathy
  • Nonaka Dis
    Date: 2013-02-04
Recruiting Study Name: 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
Condition:
  • Hereditary Inclusion Body Myopathy
  • Distal Myopathy With Rimmed
    Date: 2016-03-24
    Interventions: Drug: Aceneuramic Acid Extended-Release Tablets Other Names:
    • Ace-ER
Completed Study Name: 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
Condition:
  • GNE Myopathy
  • Hereditary Inclusion Body Myopathy

Date: 2012-01-17
Interventions:
  • Drug: Sialic Acid Extended Release (SA-ER)

Completed Study Name: Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
Condition:
  • Hereditary Inclusion Body Myopathy (HIBM)
  • GNE Myopathy

Date: 2012-07-03
Interventions: Drug: ManNAc Single dose
Recruiting Study Name: A Natural History Study of Patients With GNE Myopathy
Condition: Hereditary Inclusion Body Myopathy
Date: 2011-08-13
Completed Study Name: Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
Condition: Hereditary Inclusion Body Myopathy (HIBM)
Date: 2011-05-20
Interventions:
  • Drug: Sialic Acid Extended Release (SA-ER) Tablets

Recruiting Study Name: Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders
Condition:
  • Amyotrophic Lateral Sclerosis
  • Frontotemporal Dementia
  • PLS<
    Date: 2011-10-21
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