Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study

Article ID	Journal	Published Year	Pages	File Type
4167171	The Journal of Pediatrics	2009	9 Pages	PDF

Abstract

Sapropterin is effective in increasing Phe tolerance while maintaining blood Phe control and has an acceptable safety profile in this population of children with PKU.

Keywords

PAH PHE PKU SAE phenylketonuria intelligence quotient analysis of variance ANOVA SOC adverse event Serious adverse event Phenylalanine hydroxylase Phenylalanine

Related Topics

Health Sciences Medicine and Dentistry Perinatology, Pediatrics and Child Health

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Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study

Authors

Friedrich K. MD, Barbara K. MD, Nicola MD, PhD, Mercedes Martinez-Pardo MD, Daniel J. MD, Alex MD, Emil D. MD, PhD, Eric A. MD, Dorothy K. MD, Paul MD, Mark H. MD, Andrzej MD, PhD, Linda Marie MD, Jerry MD, PhD, Chester B. MD, PhD, Jon A. MD, Judith ScD,