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

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 anaplerotic effe...

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Detalles Bibliográficos
Autores principales:	Zöggeler, Thomas, Stock, Katharina, Jörg-Streller, Monika, Spenger, Johannes, Konstantopoulou, Vassiliki, Hufgard-Leitner, Miriam, Scholl-Bürgi, Sabine, Karall, Daniela
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2021
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7807521/ https://www.ncbi.nlm.nih.gov/pubmed/33446227 http://dx.doi.org/10.1186/s13023-020-01635-x

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