A benefit-risk analysis of pegvaliase for the treatment of phenylketonuria: A study of patients' preferences

BACKGROUND: Phenylketonuria (PKU) leads to an accumulation of phenylalanine (Phe) in the blood and subsequent neurologic, cognitive, psychiatric, and behavioral dysfunction. Many patients report social isolation and decreased quality of life. Pegvaliase is an enzyme substitution therapy that reduces...

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Detalles Bibliográficos
Autores principales: Sri Bhashyam, Sumitra, Marsh, Kevin, Quartel, Adrian, Weng, Haoling H., Gershman, Ari, Longo, Nicola, Thomas, Janet, Zori, Roberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6722251/
https://www.ncbi.nlm.nih.gov/pubmed/31497506
http://dx.doi.org/10.1016/j.ymgmr.2019.100507

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6722251/
https://www.ncbi.nlm.nih.gov/pubmed/31497506
http://dx.doi.org/10.1016/j.ymgmr.2019.100507

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