National PKU Alliance

Recent papers from National PKU Alliance

1. Efficacy and safety of sepiapterin versus sapropterin in people living with phenylketonuria: a plain language summary of the AMPLIPHY trialJournal of Comparative Effectiveness Research · 2026
2. Sepiapterin as a treatment for people living with phenylketonuria: a plain language summary of the APHENITY trialJournal of Comparative Effectiveness Research · 2025
3. The PKU Patient Registry: Development of a patient-driven registry and initial outcomesMolecular Genetics and Metabolism · 2025
4. Patient Satisfaction Following a Session With a Kidney Genetic CounselorKidney International Reports · 2025
5. Phenylketonuria (PKU): A problem solved?Molecular Genetics and Metabolism Reports · 2015
6. Newborn screening 50 years later: access issues faced by adults with PKUGenetics in Medicine · 2013