Therapeutic potential of triheptanoin in metabolic and neurodegenerative diseases.

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Wehbe Z, Tucci S

Therapeutic potential of triheptanoin in metabolic and neurodegenerative diseases.

J Inherit Metab Dis. 2020 May;43(3):385-391. doi: 10.1002/jimd.12199. Epub 2019 Dec 12.

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31778232 [ View in PubMed
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Abstract

In the past 15 years the potential of triheptanoin for the treatment of several human diseases in the area of clinical nutrition has grown considerably. Use of this triglyceride of the odd-chain fatty acid heptanoate has been proposed and applied for the treatment of several conditions in which the energy supply from citric acid cycle intermediates or fatty acid degradation are impaired. Neurological diseases due to disturbed glucose metabolism or metabolic diseases associated with impaired beta-oxidation of long chain fatty acid may especially take advantage of alternative substrate sources offered by the secondary metabolites of triheptanoin. Epilepsy due to deficiency of the GLUT1 transporter, as well as diseases associated with dysregulation of neuronal signalling, have been treated with triheptanoin supplementation, and very recently the advantages of this oil in long-chain fatty acid oxidation disorders have been reported. The present review summarises the published literature on the metabolism of triheptanoin including clinical reports related to the use of triheptanoin.

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