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Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study

Sapropterin DihydrochlorideMedicineInherited Metabolic DiseaseClinical NutritionPharmacologyPhase IiiChronic Kidney DiseaseNephrologyIncreasing Phenylalanine Tolerance