Long-term experience with triheptanoin in 12 Austrian patients with long-chain fatty acid oxidation disorders

Long-term experience with triheptanoin in 12 Austrian patients with long-chain fatty acid oxidation disorders

Abstract Background Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of rare inborn errors of metabolism with autosomal recessive inheritance that may cause life-threatening events. Treatment with triheptanoin, a synthetic seven-carbon fatty acid triglyceride compound with an anaplero...

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Bibliographic Details
Main Authors: Thomas Zöggeler, Katharina Stock, Monika Jörg-Streller, Johannes Spenger, Vassiliki Konstantopoulou, Miriam Hufgard-Leitner, Sabine Scholl-Bürgi, Daniela Karall
Format: Article
Language:English
Published: BMC 2021-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Inherited metabolic disorders
Inborn errors of metabolism
Anaplerotic effect
Fat intake regimen
Long-chain ß-oxidation disorders
Online Access:https://doi.org/10.1186/s13023-020-01635-x

Internet

https://doi.org/10.1186/s13023-020-01635-x

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