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Phase 3 Study of Sialic Acid Extended-Release Tablets in GNE Myopathy / Hereditary Inclusion Body Myopathy
2014-005432-33Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Apr 2016
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in GNE myopathy (HIBM) comparing SA-ER 6 g/day with Placebo (2 arms). Primary endpoint: Change from baseline in Upper Extremity Composite (UEC) score. Started Apr 2016; status: completed.
02
Drugs
aceneuramic acid
03
Conditions
GNE myopathy (HIBM)
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