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