Skip to main content

Advertisement

Table 2 Questions for the evaluation of sapropterin treatment

From: Diagnostic and therapeutic recommendations for the treatment of hyperphenylalaninemia in patients 0–4 years of age

• Is the Phe tolerance higher than expected based on the baseline blood Phe concentrations and the genetic test results?
• How much natural protein can the patient consume without blood Phe levels reaching above 360 μmol/L?
• Does the patient require a Phe-free amino acid mixture to cover the recommended age-appropriate protein intake?
• Is the patient tolerating the medication?