Synonyms: Dojolvi® | UX-007 | UX007
triheptanoin is an approved drug (FDA (2020))
Compound class:
Synthetic organic
Comment: Triheptanoin is a medium-chain triglyceride that is administered as a rescue therapeutic in patients with long-chain fatty acid oxidation disorders [1].
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References |
1. Shirley M. (2020)
Triheptanoin: First Approval. Drugs, 80 (15): 1595-1600. [PMID:32897506] |
2. Vockley J, Burton B, Berry G, Longo N, Phillips J, Sanchez-Valle A, Chapman K, Tanpaiboon P, Grunewald S, Murphy E et al.. (2021)
Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study. J Inherit Metab Dis, 44 (1): 253-263. [PMID:32885845] |
3. Vockley J, Burton B, Berry GT, Longo N, Phillips J, Sanchez-Valle A, Tanpaiboon P, Grunewald S, Murphy E, Bowden A et al.. (2019)
Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD). J Inherit Metab Dis, 42 (1): 169-177. [PMID:30740733] |