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Real-world treatment, dosing, and discontinuation patterns among patients treated with pegvaliase for phenylketonuria: Evidence from dispensing data

BACKGROUND: Phenylketonuria (PKU) is an inborn metabolic error characterized by a deficiency of the enzyme required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods. Pegvaliase (Palynziq®) is an enzyme substitution therapy approved for adults with P...

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Detalles Bibliográficos
Autores principales: Lah, Melissa, Cook, Keziah, Gomes, Dumingu Aparna, Liu, Stephanie, Tabatabaeepour, Nadia, Kirson, Noam, Chen, Er, Lindstrom, Kristin, Whitehall, Kaleigh Bulloch, Van Backle, Joost, Burton, Barbara K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9513695/
https://www.ncbi.nlm.nih.gov/pubmed/36176956
http://dx.doi.org/10.1016/j.ymgmr.2022.100918