6 studies found for:    MYOPATHY, DISTAL, 2
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Rank Status Study
1 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
2 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:
3 Completed Deflazacort in Dysferlinopathies
Conditions: LGMD2B;   Miyoshi Myopathy;   Dysferlinopathy
Interventions: Drug: deflazacort;   Drug: placebo
4 Active, not recruiting Clinical Outcome Study for Dysferlinopathy
Conditions: Dysferlinopathy;   LGMD2B;   Miyoshi Myopathy
Intervention:
5 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
6 Recruiting A Natural History Study of Patients With Hereditary Inclusion Body Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention:

Indicates status has not been verified in more than two years