Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2 months of age with urea cycle disorders

BACKGROUND/AIMS: Neonatal onset Urea cycle disorders (UCDs) can be life threatening with severe hyperammonemia and poor neurological outcomes. Glycerol phenylbutyrate (GPB) is safe and effective in reducing ammonia levels in patients with UCD above 2 months of age. This study assesses safety, ammoni...

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Detalles Bibliográficos
Autores principales: Longo, Nicola, Diaz, George A., Lichter-Konecki, Uta, Schulze, Andreas, Inbar-Feigenberg, Michal, Conway, Robert L., Bannick, Allison A., McCandless, Shawn E., Zori, Roberto, Hainline, Bryan, Mew, Nicholas Ah, Canavan, Colleen, Vescio, Thomas, Kok, Teresa, Porter, Marty H., Berry, Susan A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8655853/
https://www.ncbi.nlm.nih.gov/pubmed/33388234
http://dx.doi.org/10.1016/j.ymgme.2020.12.002

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