Please use this identifier to cite or link to this item: https://hdl.handle.net/20.500.12530/27009
Title: Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study.
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Issue Date: 31-Mar-2016
Citation: Orphanet J Rare Dis.2016 Mar;(11):32
Abstract: 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.
PMID: 27030250
URI: https://hdl.handle.net/20.500.12530/27009
Rights: openAccess
Appears in Collections:Fundaciones e Institutos de Investigación > IIS H. U. La Paz > Artículos
Fundaciones e Institutos de Investigación > IIS H. U. Ramón y Cajal > Artículos
Hospitales > H. U. La Paz > Artículos

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