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Carglumic acid: an additional therapy in the treatment of organic acidurias with hyperammonemia?

BACKGROUND: Hyperammonemia in patients with methylmalonic aciduria (MMA) and propionic aciduria (PA) is caused by accumulation of propionyl-CoA which decreases the synthesis of N-acetyl-glutamate, the natural activator of carbamyl phosphate synthetase 1. A treatment approach with carglumic acid, the...

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Главные авторы: Levrat, Virginie, Forest, Isabelle, Fouilhoux, Alain, Acquaviva, Cécile, Vianey-Saban, Christine, Guffon, Nathalie
Формат: Artigo
Язык:Inglês
Опубликовано: BioMed Central 2008
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC2262878/
https://ncbi.nlm.nih.gov/pubmed/18234091
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-3-2
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