Clinical Trial: Study to Evaluate the Safety and Efficacy of Ace-ER Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional




Official Title: 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

Brief Summary: This open-label extension study of Ace-ER will assess the long-term safety and efficacy of Ace-ER treatment over a period of 24 months. Approximately 165 subjects from the UX001-CL202, UX001-CL301 and UX001-CL203 studies will be eligible to enroll in the study.