
Contents
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Introduction to Anaplerosis and Triheptanoin Introduction to Anaplerosis and Triheptanoin
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Energy Metabolism in Epilepsy Energy Metabolism in Epilepsy
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Triheptanoin Alters Brain Energy Metabolism in Various Settings, Including Epilepsy Models Triheptanoin Alters Brain Energy Metabolism in Various Settings, Including Epilepsy Models
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Anticonvulsant Effects of Triheptanoin and Clinical Trials Anticonvulsant Effects of Triheptanoin and Clinical Trials
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Triheptanoin and Other Disorders Triheptanoin and Other Disorders
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Metabolic Disorders: Glucose Transporter Type 1 Deficiency, Long Chain Fatty Acid Oxidation Disorders, Pyruvate Decarboxylase Deficiency Metabolic Disorders: Glucose Transporter Type 1 Deficiency, Long Chain Fatty Acid Oxidation Disorders, Pyruvate Decarboxylase Deficiency
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Other Disorders: Neurological Conditions, Muscle Disorders, Cardiac Hypertrophy Other Disorders: Neurological Conditions, Muscle Disorders, Cardiac Hypertrophy
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Conclusions Conclusions
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Disclosure/Conflict of Interest Disclosure/Conflict of Interest
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Acknowledgments Acknowledgments
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References References
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34 Triheptanoin in Epilepsy and Beyond
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Published:October 2016
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Abstract
Triheptanoin, the triglyceride of heptanoate (C7 fatty acid), is a novel treatment that is being used to treat patients with rare genetic metabolic disorders. When taken orally, triheptanoin is hydrolyzed in the gastrointestinal tract to heptanoate, which is thought to diffuse into the blood and body. Heptanoate and its liver ketone metabolites are then metabolized within cells to propionyl-CoA, which after carboxylation produces succinyl-CoA, resulting in anaplerosis—the refilling of a deficient tricarboxylic acid cycle. Here, data are summarized and discussed in relation to triheptanoin’s anticonvulsant effects in rodent seizure models. Biochemical data reveal that metabolic alterations found in brains of rodent seizure models can be restored by triheptanoin. Moreover, there are increasing preclinical and clinical studies indicating that triheptanoin is beneficial in other neurological and neuromuscular disorders, which are summarized here. Thus, triheptanoin seems to be a promising treatment for a variety of clinical conditions.
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