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Phase 2 open-label UX007 in long-chain fatty acid oxidation disorders
2013-004830-14Phase 2Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Feb 2014
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of triheptanoin in long-chain fatty acid oxidation disorder (LC-FAOD), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Clinical effects of UX007 on LC-FAOD pathophysiology. Started Feb 2014; status: completed.
02
Drugs
triheptanoin
03
Conditions
long-chain fatty acid oxidation disorder (LC-FAOD)
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