Clinical experience with N-carbamylglutamate in a single-centre cohort of patients with propionic and methylmalonic aciduria

BACKGROUND: The effect of long-term N-carbamylglutamate (NCG) treatment on the rate and severity of decompensations due to propionic aciduria (PA) and methylmalonic aciduria (MMA) is unknown. This paper presents clinical experience from a single-centre cohort of patients with PA and MMA who received...

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Publicat a:Mol Genet Metab Rep
Autors principals: Burlina, Alberto, Cazzorla, Chiara, Zanonato, Elisa, Viggiano, Emanuela, Fasan, Ilaria, Polo, Giulia
Format: Artigo
Idioma:Inglês
Publicat: Elsevier 2016
Matèries:
Research Paper
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4949587/
https://ncbi.nlm.nih.gov/pubmed/27489777
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2016.06.007
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