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3 studies found for:    MYOPATHY, DISTAL, 2
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MYOPATHY, DISTAL, 2 (3 records)
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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 Recruiting A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment
Conditions: Hereditary Inclusion Body Myopathy;   Distal Myopathy With Rimmed Vacuoles;   Distal Myopathy, Nonaka Type;   GNE Myopathy;   Quadriceps Sparing Myopathy;   Inclusion Body Myopathy 2
Intervention: Drug: Aceneuramic Acid Extended-Release Tablets
3 Recruiting A Natural History Study of Patients With GNE Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention:

Study has passed its completion date and status has not been verified in more than two years.