Is sapropterin treatment suitable for all subjects with phenylketonuria?


In a Phase I study, 20% of subjects with phenylketonuria (96/485) responded with a > or = 30% reduction in blood phenylalanine at day 8 of treatment with sapropterin. Subsequently, some of the responders from this study were enrolled in a Phase III study. The subjects with phenylketonuria were randomised to sapropterin 10 mg/kg/day (n = 41) or placebo (n… (More)


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