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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Publicat a:Clin Pharmacokinet
Autors principals: Qi, Yulan, Mould, Diane R., Zhou, Huiyu, Merilainen, Markus, Musson, Donald G.
Format: Artigo
Idioma:Inglês
Publicat: Springer International Publishing 2014
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Original Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4306193/
https://ncbi.nlm.nih.gov/pubmed/25338975
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40262-014-0196-4
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