Effect of carglumic acid with or without ammonia scavengers on hyperammonaemia in acute decompensation episodes of organic acidurias

Abstract Background Hyperammonaemia is a key sign of decompensation in organic acidurias (OAs) and can contribute to severe neurological complications, thus requiring rapid treatment. Methods A post-hoc analysis of two retrospective studies analysed the efficacy of carglumic acid ± ammonia (NH3) sca...

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Bibliographic Details
Main Authors: Anupam Chakrapani, Vassili Valayannopoulos, Nuria García Segarra, Mireia Del Toro, Maria Alice Donati, Angeles García-Cazorla, María Julieta González, Celine Plisson, Vincenzo Giordano
Format: Article
Language:English
Published: BMC 2018-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Online Access:http://link.springer.com/article/10.1186/s13023-018-0840-4