Table 3.
Summary of treatment and reimbursement information (N = 31)
| What treatment options are available in your country? | |||||
| Low-Phe diet | BH4 | Large neutral amino acids | Othera | ||
| 100 % | 48.4 % | 32.3 % | 3.2 % | ||
| At what age does your centre introduce low-Phe diet in newly diagnosed newborns? | |||||
| 0–9 days | 10–15 days | 16–28 days | ≥29 days | No answer | |
| 19.4 % | 51.6 % | 22.6 % | 3.2 % | 3.2 % | |
| Who contributes to the costs of Phe-free protein substitutes?b | |||||
| Government | Private health insurance | Parents/patients | |||
| 96.8 % | 12.9 % | 22.6 % | |||
| Who contributes to the cost of special low-protein foods (flour, pasta)?b | |||||
| Government | Private health insurance | Parents/patients | Otherc | ||
| 59.4 % | 12.5 % | 75.0 % | 6.3 % | ||
| What kind of social support is offered to PKU patients in your country?b | |||||
| Disability allowance | Disability certificate which helps with education, employment, travel expenses | Dietary allowance to go to summer camps | Reimbursement of travel expenses | Home support or compensation to parents for decreasing working hours | No support available |
| 80.6 % | 45.2 % | 32.3 % | 25.8 % | 12.9 % | 9.7 % |
BH4 sapropterin dihydrochoride, Phe phenylalanine, PKU phenylketonuria
a‘Other’ was specified as ‘BH4 only for BH4-deficient patients’
bMultiple answers were possible, and therefore, the total exceeds 100 %
c‘Other’ was specified as: ‘sellers’, ‘non-governmental organisation’ and ‘parents/patients association’