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Dietary management and major clinical events in patients with longchain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study

BACKGROUND & AIMS: Long-chain fatty acid oxidation disorders (LC-FAOD) are rare, life-threatening, autosomal recessive disorders that lead to energy depletion and major clinical events (MCEs), such as acute metabolic crises of hypoglycemia, cardiomyopathy, and rhabdomyolysis. The aim of this stu...

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Detalles Bibliográficos
Autores principales: Vockley, Jerry, Longo, Nicola, Madden, Megan, Dwyer, Lauren, Mu, Yunming, Chen, Chao-Yin, Cataldo, Jason
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8567087/
https://www.ncbi.nlm.nih.gov/pubmed/33487279
http://dx.doi.org/10.1016/j.clnesp.2020.11.018