Background: Isovaleric aciduria (IVA), propionic aciduria (PA) and methylmalonic aciduria (MMA) are inherited organic acidurias (OAs) in which impaired organic acid metabolism induces hyperammonaemia arising partly from secondary deficiency of N-acetylglutamate (NAG) synthase. Rapid reduction in plasma ammonia is required to prevent neurological complications. This retrospective, multicentre, open-label, uncontrolled, phase IIIb study evaluated the efficacy and safety of carglumic acid, a synthetic structural analogue of NAG, for treating hyperammonaemia during OA decompensation.
View Article and Find Full Text PDFBackground: Advances in the diagnosis and treatment of urea cycle disorders (UCDs) have led to a higher survival rate. The purpose of this study is to describe the characteristics of patients with urea cycle disorders in Spain.
Methods: Observational, cross-sectional and multicenter study.
Unlabelled: Malnutrition among hospitalized patients has clinical implications and is associated with adverse outcomes: depression of the immune system, impaired wound healing, muscle wasting, longer length of stay, higher costs and increased mortality. Although the rate of malnutrition in hospitalized children varies in different studies, it seems to be lower than in adult population. Nevertheless, this is a population that has a higher risk of developing malnutrition during hospital stay.
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