10 studies found for:    "inclusion body myopathy 2"
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Rank Status Study
1 Recruiting Hereditary Inclusion Body Myopathy-Disease Monitoring (HIBM-DMP): A Combined Registry and Prospective Observational Natural History Study to Assess HIBM Disease
Conditions: Hereditary Inclusion Body Myopathy;   GNE Myopathy;   Nonaka Disease;   Quadriceps Sparing Myopathy (QSM);   Distal Myopathy With Rimmed Vacuoles (DMRV)
Intervention:
2 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
3 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
4 Completed Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention: Drug: Immune Globulin
5 Recruiting A Natural History Study of Patients With Hereditary Inclusion Body Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention:
6 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
7 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
8 Completed Pharmacokinetic Study on N-acetylneuraminic Acid
Conditions: Nonaka Myopathy;   Hereditary Inclusion Body Myopathy
Intervention: Drug: NPC-09
9 Enrolling by invitation 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
10 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