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 - Category: Research Source Type: clinical trials