14 studies found for:    NONAKA MYOPATHY
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Rank Status Study
1 Recruiting 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)
Conditions: Hereditary Inclusion Body Myopathy;   GNE Myopathy;   Nonaka Disease;   Quadriceps Sparing Myopathy (QSM);   Distal Myopathy With Rimmed Vacuoles (DMRV)
Intervention:
2 Recruiting 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
Conditions: Hereditary Inclusion Body Myopathy;   Distal Myopathy With Rimmed Vacuoles;   Distal Myopathy, Nonaka Type;   GNE Myopathy;   Quadriceps Sparing Myopathy;   Inclusion Body Myopathy 2
Intervention: Drug: Aceneuramic Acid Extended-Release Tablets
3 Enrolling by invitation Study to Evaluate the Safety and Efficacy of Ace-ER Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
Conditions: Hereditary Inclusion Body Myopathy;   Distal Myopathy With Rimmed Vacuoles;   Distal Myopathy, Nonaka Type;   GNE Myopathy;   Quadriceps Sparing Myopathy
Intervention: Drug: Aceneuramic Acid Extended-Release Tablets
4 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)
Conditions: Hereditary Inclusion Body Myopathy;   Distal Myopathy With Rimmed Vacuoles;   Distal Myopathy, Nonaka Type
Interventions: Drug: Sialic Acid Tablets;   Drug: Placebo Tablets
5 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
Conditions: GNE Myopathy;   Hereditary Inclusion Body Myopathy
Interventions: Drug: Sialic Acid Extended Release (SA-ER);   Drug: Placebo
6 Completed Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
Conditions: Hereditary Inclusion Body Myopathy (HIBM);   GNE Myopathy
Intervention: Drug: ManNAc
7 Completed Pharmacokinetic Study on N-acetylneuraminic Acid
Conditions: Nonaka Myopathy;   Hereditary Inclusion Body Myopathy
Intervention: Drug: NPC-09
8 Active, not recruiting An Open Label Phase 2 Study of DEX-M74 in Subjects With GNE Myopathy
Condition: GNE Myopathy
Interventions: Drug: ManNac.;   Drug: ManNac
9 Recruiting A Natural History Study of Patients With GNE Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention:
10 Completed Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention: Drug: Immune Globulin
11 Active, not recruiting An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid (ER Tablets + IR Capsules) in Patients With GNE Myopathy
Conditions: GNE Myopathy;   HIBM
Intervention: Drug: SA-ER tablets and SA-IR capsules
12 Completed Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
Condition: Hereditary Inclusion Body Myopathy (HIBM)
Interventions: Drug: Sialic Acid Extended Release (SA-ER) Tablets;   Drug: Sialic Acid Extended Release (SA-ER) Tables
13 Completed Natural History in CCFDN and IBM Syndromes
Conditions: Inclusion Body Myositis, Sporadic;   Inclusion Body Myopathy, Autosomal-recessive;   Inclusion Body Myopathy, Autosomal-dominant;   Congenital Cataracts, Facial Dysmorphism, And Neuropathy
Intervention: Other: Natural History
14 Active, not recruiting Clinical, Biological and NMR Outcome Measures Study for Hereditary Inclusion Body Myopathy Due to Mutation of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine Kinase Gene (GNE)
Condition: HIBM
Interventions: Other: motor function and strength assessment;   Other: NMR assessment;   Other: 24h urine and serum collection

Indicates status has not been verified in more than two years