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PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: a phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study

Drago Bratkovic, Lali Margvelashvili, Michel Tchan, Janelle Nisbet, Neil Smith

2021Metabolism47 citationsDOIOpen Access PDF

Topics & Concepts

Phenylalanine hydroxylaseTetrahydrobiopterinHyperphenylalaninemiaCrossover studyPhenylalanineWashoutInternal medicineMedicinePhenylketonuriasChemistryEndocrinologyAmino acidBiochemistryPlaceboNitric oxide synthasePathologyAlternative medicineNitric oxideMetabolism and Genetic DisordersPharmacogenetics and Drug MetabolismDiet and metabolism studies
PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: a phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study | Litcius