Efficacy of bezafibrate in two patients with mitochondrial trifunctional protein deficiency

Efficacy of bezafibrate in two patients with mitochondrial trifunctional protein deficiency

Mitochondrial trifunctional protein (TFP) deficiency is a rare inherited metabolic disorder caused by defects in fatty acid β-oxidation (FAO) of long-chain fatty acids, leading to impaired energy production. Fasting avoidance, fatty acid-restricted diets, and supplementation with medium-chain trigly...

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Bibliographic Details
Main Authors: Tomonori Suyama, Masaru Shimura, Takuya Fushimi, Naomi Kuranobu, Keiko Ichimoto, Ayako Matsunaga, Masaki Takayanagi, Kei Murayama
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Bezafibrate
TFP deficiency
Myalgia
Rhabdomyolysis
Fatty acid β-oxidation disorders (FAODs)
l-carnitine
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426920300562

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http://www.sciencedirect.com/science/article/pii/S2214426920300562

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