A Prospective Population Pharmacokinetic Analysis of Sapropterin Dihydrochloride in Infants and Young Children with Phenylketonuria

BACKGROUND AND OBJECTIVES: Untreated phenylketonuria (PKU), a hereditary metabolic disorder caused by a genetic mutation in phenylalanine hydroxylase (PAH), is characterized by elevated blood phenylalanine (Phe) and severe neurologic disease. Sapropterin dihydrochloride, a synthetic preparation of n...

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Detalles Bibliográficos
Autores principales: Qi, Yulan, Mould, Diane R., Zhou, Huiyu, Merilainen, Markus, Musson, Donald G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2014
Materias:
Original Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4306193/
https://www.ncbi.nlm.nih.gov/pubmed/25338975
http://dx.doi.org/10.1007/s40262-014-0196-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4306193/
https://www.ncbi.nlm.nih.gov/pubmed/25338975
http://dx.doi.org/10.1007/s40262-014-0196-4

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