Overview

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)

Status:
Terminated
Trial end date:
2018-01-10
Target enrollment:
Participant gender:
Summary
The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.
Phase:
Phase 3
Details
Lead Sponsor:
Ultragenyx Pharmaceutical Inc