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Ace-ER Safety in Severe GNE Myopathy
NCT02731690Phase 2Terminated
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Apr 2016
- Primary completion
- Jan 2018
- Modality
- Small Molecules
- Countries
- 3
01
Overview
Phase 2, open-label, single-arm trial of aceneuramic acid in GNE myopathy (HIBM), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Treatment-Emergent Adverse Events at 48 weeks. Started Apr 2016; status: terminated.
02
Drugs
aceneuramic acid
03
Conditions
GNE myopathy (HIBM)
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