Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients

Key European guidelines for the diagnosis and management of patients with phenylketonuria

(2017) Francjan J van Spronsen et al.   Lancet Diabetes & Endocrinology

Comparison of tandem mass spectrometry and amino acid analyzer for phenylalanine and tyrosine monitoring—Implications for clinical management of patients with hyperphenylalaninemia

(2015) Urh Groselj et al.   CLINICAL BIOCHEMISTRY

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

(2014) Yulan Qi et al.   CLINICAL PHARMACOKINETICS

Long-term developmental progression in infants and young children taking sapropterin for phenylketonuria: a two-year analysis of safety and efficacy

(2014) Nicola Longo et al.   GENETICS IN MEDICINE

Sapropterin Is Safe and Effective in Patients less than 4-Years-Old with BH4-Responsive Phenylalanine Hydroxylase Deficiency

(2014) Haruo Shintaku et al.   JOURNAL OF PEDIATRICS

Phenylketonuria Scientific Review Conference: State of the science and future research needs

(2014) Kathryn M. Camp et al.   MOLECULAR GENETICS AND METABOLISM

Simultaneous determination of phenylalanine and tyrosine in peripheral capillary blood by HPLC with ultraviolet detection

(2013) Xi-ming Mo et al.   CLINICAL BIOCHEMISTRY

Phenylketonuria: nutritional advances and challenges

(2012) Marcello Giovannini et al.   Nutrition & Metabolism

Diagnosis, classification, and genetics of phenylketonuria and tetrahydrobiopterin (BH4) deficiencies

(2011) Nenad Blau et al.   MOLECULAR GENETICS AND METABOLISM

Phenylketonuria

(2010) Nenad Blau et al.   LANCET

Sapropterin

(2009) Mark Sanford et al.   DRUGS

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

(2009) Friedrich K. Trefz et al.   JOURNAL OF PEDIATRICS

Reassessment of phenylalanine tolerance in adults with phenylketonuria is needed as body mass changes

(2009) Erin L. MacLeod et al.   MOLECULAR GENETICS AND METABOLISM

Pharmacokinetics of Sapropterin in Patients with Phenylketonuria

(2008) François Feillet et al.   CLINICAL PHARMACOKINETICS

Effect of BH4 supplementation on phenylalanine tolerance

(2008) A. Burlina et al.   JOURNAL OF INHERITED METABOLIC DISEASE