Woman and Man
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Extract
The primary objective of the study is to evaluate the proportion of responders (≥30% reduction from baseline in blood Phe level) to 20 mg/kg/day Sapropterin dihydrochloride treatment at several time points during 28 +/- 1 days.
Inclusion criteria
- Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA))