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Ace-ER open-label extension in GNE myopathy
2016-000360-42Phase 3Terminated
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- May 2016
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of aceneuramic acid in GNE myopathy (HIBM), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Overall Safety Endpoint. Started May 2016; status: terminated.
02
Drugs
aceneuramic acid
03
Conditions
GNE myopathy (HIBM)
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