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triheptanoin + medium-chain triglycerides

Development stage: Unknown
Lead developer: Ultragenyx Pharmaceutical
Modality: Small Molecules
Administration: Oral

Overview

Triheptanoin (Oil special 107) is an odd-chain medium-chain triglyceride (C7) that serves as an anaplerotic agent by providing propionyl-CoA, which can be converted to succinyl-CoA to replenish the citric acid cycle. Mygliol 810 is a standard even-chain medium-chain triglyceride (MCT) oil (C8/C10) used as a caloric source and comparator. This combination/regimen was investigated by Assistance Publique - Hôpitaux de Paris for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), where patients cannot effectively metabolize long-chain fats. The trial aimed to determine if the anaplerotic effects of triheptanoin provided superior clinical outcomes compared to conventional MCT therapy by bypassing metabolic blocks and providing alternative energy substrates.

Brand names: Spezialöl 107Migliol 810Migliol810Migliol-810Dojolvi
Other names: TriheptanoinMCT oilOdd-chain medium-chain triglyceridesEven-chain medium-chain triglyceridesTri-C7Tri-C-7Tri-C 7

Targets

Mitochondrial beta-oxidation enzymes

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