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7 studies found for:    "distal myopathy 2" OR "Distal Myopathies"

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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 Deflazacort in Dysferlinopathies
Conditions: LGMD2B;   Miyoshi Myopathy;   Dysferlinopathy
Interventions: Drug: deflazacort;   Drug: placebo
4 Active, not recruiting 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:
5 Recruiting Clinical Outcome Study for Dysferlinopathy
Conditions: Dysferlinopathy;   LGMD2B;   Miyoshi Myopathy
Intervention:
6 Unknown † The Efficacy of Using Far Infrared Radiation to Manage Muscular Dystrophies
Condition: Muscular Dystrophies
Intervention: Radiation: Far Infrared Radiation
7 Active, not recruiting 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
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† Indicates status has not been verified in more than two years