Long-term comparative effectiveness of pegvaliase versus medical nutrition therapy with and without sapropterin in adults with phenylketonuria

This publication has 25 references indexed in Scilit:

• A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria
  Molecular Genetics and Metabolism, 2015
• Phenylalanine hydroxylase deficiency: diagnosis and management guideline
  Genetics in Medicine, 2014
• Adherence Issues in Inherited Metabolic Disorders Treated by Low Natural Protein Diets
  Annals of Nutrition and Metabolism, 2012
• Are propensity scores really superior to standard multivariable analysis?
  Contemporary Clinical Trials, 2011
• Problems with Using the Normal Distribution – and Ways to Improve Quality and Efficiency of Data Analysis
  PLOS ONE, 2011
• Blood phenylalanine control in phenylketonuria: a survey of 10 European centres
  European Journal of Clinical Nutrition, 2010
• The reality of dietary compliance in the management of phenylketonuria
  Journal of Inherited Metabolic Disease, 2010
• Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study
  The Journal of Pediatrics, 2009
• The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R‐tetrahydrobiopterin): a phase II, multicentre, open‐label, screening study
  Journal of Inherited Metabolic Disease, 2007
• How practical are recommendations for dietary control in phenylketonuria?
  The Lancet, 2002