12 studies found for:    "Distal Myopathies" OR "Laing distal myopathy"
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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 Pharmacokinetic Study on N-acetylneuraminic Acid
Conditions: Nonaka Myopathy;   Hereditary Inclusion Body Myopathy
Intervention: Drug: NPC-09
3 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
4 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
5 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
6 Completed Deflazacort in Dysferlinopathies
Conditions: LGMD2B;   Miyoshi Myopathy;   Dysferlinopathy
Interventions: Drug: deflazacort;   Drug: placebo
7 Recruiting A Natural History Study of Patients With Hereditary Inclusion Body Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention:
8 Completed Evaluation of Limb-Girdle Muscular Dystrophy
Conditions: Becker Muscular Dystrophy;   Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency);   Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency);   Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
Intervention:
9 Completed Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention: Drug: Immune Globulin
10 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
11 Active, not recruiting Clinical Outcome Study for Dysferlinopathy
Conditions: Dysferlinopathy;   LGMD2B;   Miyoshi Myopathy
Intervention:
12 Unknown  The Efficacy of Using Far Infrared Radiation to Manage Muscular Dystrophies
Condition: Muscular Dystrophies
Intervention: Radiation: Far Infrared Radiation

Indicates status has not been verified in more than two years