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Long-term comparative effectiveness of pegvaliase versus standard of care comparators in adults with phenylketonuria

Phenylketonuria (PKU) is caused by phenylalanine hydroxylase (PAH) deficiency, resulting in high blood and brain Phenylalanine (Phe) concentrations that can lead to impaired brain development and function. Standard treatment involves a Phe-restricted diet alone or in conjunction with sapropterin dih...

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Detalles Bibliográficos
Autores principales:	Zori, Roberto, Ahring, Kirsten, Burton, Barbara, Pastores, Gregory M., Rutsch, Frank, Jha, Ashok, Jurecki, Elaina, Rowell, Richard, Harding, Cary
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9013411/ https://www.ncbi.nlm.nih.gov/pubmed/31439512 http://dx.doi.org/10.1016/j.ymgme.2019.07.018

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