30. 遠位型ミオパチー Distal myopathy Clinical trials / Disease details


臨床試験数 : 15 薬物数 : 17 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 1

  
1 trial found
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agemin
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PhaseCountries
1NCT02736188
(ClinicalTrials.gov)
May 2, 20164/4/2016Study 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 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type;GNE Myopathy;Quadriceps Sparing MyopathyDrug: Aceneuramic Acid Extended-Release TabletsUltragenyx Pharmaceutical IncNULLTerminated18 YearsN/AAll143Phase 3United States;Bulgaria;Canada;France;Israel;Italy;United Kingdom