12 studies found for:    "distal myopathy 2" OR "Distal Myopathies"
Show Display Options
RSS Create an RSS feed from your search for:
"distal myopathy 2" OR "Distal Myopathies"
Need help? See RSS Feeds
Choose a feed type:
Show studies first received on any dateShow studies that were first received in the last 14 days
Show studies last updated date on any dateShow studies that were added or modified in the last 14 days
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