Clinical experience with glycerol phenylbutyrate in 20 patients with urea cycle disorders at a UK paediatric centre

Clinical experience with glycerol phenylbutyrate in 20 patients with urea cycle disorders at a UK paediatric centre

Abstract In urea cycle disorders (UCDs) ammonia scavenger drugs, usually sodium‐based, have been the mainstay of treatment. Increasingly, glycerol phenylbutyrate (GPB, Ravicti®) is being used but scant real‐world data exist regarding clinical outcomes. A retrospective study of UCD patients initiated...

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Bibliographic Details
Main Authors: Mildrid Yeo, Preeya Rehsi, Megan Dorman, Stephanie Grunewald, Julien Baruteau, Anupam Chakrapani, Emma Footitt, Helen Prunty, Melanie McSweeney
Format: Article
Language:English
Published: Wiley 2023-09-01
Series:JIMD Reports
Subjects:
glycerol phenylbutyrate
hyperammonaemia
Ravicti
sodium benzoate
sodium phenylbutyrate
urea cycle disorders
Online Access:https://doi.org/10.1002/jmd2.12386

Internet

https://doi.org/10.1002/jmd2.12386

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