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

Tetrahydrobiopterin therapy vs phenylalanine-restricted diet: Impact on growth in PKU

(2013) Luis Aldámiz-Echevarría et al.   MOLECULAR GENETICS AND METABOLISM

Long-term Follow-up and Outcome of Phenylketonuria Patients on Sapropterin: A Retrospective Study

(2013) S. Keil et al.   PEDIATRICS

Efficacy and safety of BH4 before the age of 4 years in patients with mild phenylketonuria

(2012) Oriane Leuret et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Recommendations for the use of sapropterin in phenylketonuria

(2012) Amy Cunningham et al.   MOLECULAR GENETICS AND METABOLISM

Phenylalanine hydroxylase deficiency

(2011) John J. Mitchell et al.   GENETICS IN MEDICINE

Correlation of age-specific phenylalanine levels with intellectual outcome in patients with phenylketonuria

(2011) Krista S. Viau et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: Results of a phase 3b study

(2011) Barbara K. Burton et al.   MOLECULAR GENETICS AND METABOLISM

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

Outcomes beyond phenylalanine: An international perspective☆

(2009) Francois Feillet et al.   MOLECULAR GENETICS AND METABOLISM

Beyond executive function: Non-executive cognitive abilities in individuals with PKU☆

(2009) Darren Janzen et al.   MOLECULAR GENETICS AND METABOLISM

Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria

(2008) Phillip Lee et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A