Additional file 3: Table S3. of Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countries

Comparisons of PKU-QOL scores according to BH4 intake. This file includes four tables presenting in the child, adolescent, adult and parent samples the comparison of PKU-QOL scores according to BH4 intake (BH4 intake; no BH4 intake)

Additional file 2: Table S2. of Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countries

Comparisons of PKU-QOL scores according to the overall health status as assessed by the clinicians. This file includes four tables presenting in the child, adolescent, adult and parent samples the comparison of PKU-QOL scores according to the overall assessment of health status made by the clinicians (poor; fair; good; very good; excellent)

Additional file 1: Table S1. of Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countries

Comparisons of PKU-QOL scores according to the severity of PKU. This file includes four tables presenting in the child, adolescent, adult and parent samples the comparison of PKU-QOL scores according to the severity of PKU (mild-moderate PKU: Phe level 600–1200 μmol/L; Classical PKU: Phe level >1200 μmol/L)

Additional file 1: Figure S1. of Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial

Summary of neuromotor developmental milestones – ITT population. Data show (a) the proportion of patients with normal development in the area of assessment at baseline, (b) Week 12, and (c) Week 26 treated with sapropterin plus the Phe-restricted diet, or Phe-restricted diet alone. P-values show comparison of results between treatment groups at Week 26 using the chi-squared test. Table S1. PAH genotypes of sapropterin responders (n=37). (DOCX 180 kb