Trial Profile
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Status:
Completed
Phase of Trial:
Phase III
Latest Information Update: 12 Dec 2022
Price :
$35
*
At a glance
- Drugs Aceneuramic acid (Primary)
- Indications Nonaka distal myopathy
- Focus Registrational; Therapeutic Use
- Acronyms GNEM
- Sponsors Ultragenyx Pharmaceutical
- 30 Apr 2019 Results published in the Neurology
- 22 Aug 2017 According to an Ultragenyx Pharmaceutical media release, based on results of this trial Ultragenyx plans to discontinue further clinical development of Ace-ER.
- 22 Aug 2017 Results published in an Ultragenyx Pharmaceutical media release.