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)
Latest Information Update: 10 Mar 2022
At a glance
- Drugs Aceneuramic acid (Primary)
- Indications Nonaka distal myopathy
- Focus Adverse reactions; Registrational; Therapeutic Use
- Sponsors Ultragenyx Pharmaceutical
Most Recent Events
- 13 Feb 2019 New trial focus therapeutics use and new primary end point has been added.
- 01 Jun 2018 Status changed from active, no longer recruiting to discontinued.
- 28 Jan 2018 The study has been discontinued in Bulgaria.