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

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 on or sw...

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Detalles Bibliográficos
Autores principales: Yeo, Mildrid, Rehsi, Preeya, Dorman, Megan, Grunewald, Stephanie, Baruteau, Julien, Chakrapani, Anupam, Footitt, Emma, Prunty, Helen, McSweeney, Melanie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2023
Materias:
Case Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10494499/
https://www.ncbi.nlm.nih.gov/pubmed/37701329
http://dx.doi.org/10.1002/jmd2.12386

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10494499/
https://www.ncbi.nlm.nih.gov/pubmed/37701329
http://dx.doi.org/10.1002/jmd2.12386

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