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Study to Evaluate the Safety and Efficacy of Ace-ER Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
Conditions:   Hereditary Inclusion Body Myopathy;   Distal Myopathy With Rimmed Vacuoles;   Distal Myopathy, Nonaka Type;   GNE Myopathy;   Quadriceps Sparing MyopathyIntervention:   Drug: Aceneuramic Acid Extended-Release TabletsSponsor:   Ultragenyx Pharmaceutical IncEnrolling by invitation - verified April 2016 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - April 4, 2016 Category: Research Source Type: clinical trials

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 2Intervention:   Drug: Aceneuramic Acid Extended-Release TabletsSponsor:   Ultragenyx Pharmaceutical IncNot yet recruiting - verified April 2016 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - March 24, 2016 Category: Research Source Type: clinical trials