{"disease":{"id":"distal-myopathy-nonaka-type","name":"distal myopathy nonaka type"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT02736188","title":"Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)","phase":"PHASE3","overall_status":"TERMINATED","enrollment_count":143,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"nct_id":"NCT02377921","title":"Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":89,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"nct_id":"NCT02731690","title":"A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment","phase":"PHASE2","overall_status":"TERMINATED","enrollment_count":42,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true}],"total":3},"guidelines":[],"source":"Drug Landscape verified database"}