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Ace-ER in GNE Myopathy
NCT02377921Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- May 2015
- Primary completion
- Jun 2017
- Modality
- Small Molecules
- Countries
- 7
01
Overview
Phase 3, randomised, double-blind trial in GNE myopathy (HIBM) comparing Ace-ER with Placebo (2 arms). Primary endpoint: Upper Extremity Composite Score at 48 weeks. Started May 2015; status: completed.
02
Drugs
aceneuramic acid
03
Conditions
GNE myopathy (HIBM)
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