Published in The Lancet 2024, this research article presents findings from APHENITY, a Phase 3 clinical trial evaluating the efficacy and safety of sepiapterin in patients with phenylketonuria (PKU)
All publications
Browse selected scientific articles published in peer-reviewed international and interdisciplinary journals to gain insights into the clinical presentation, and genetic and molecular basis of phenylketonuria (PKU)
|
Topic
Type
Year
This is an author video developed to convey the key messages of the APHENITY trial manuscripts through the voices of two key authors
Published in Genetics in Medicine, this review paper explores the current and future management and therapeutic strategies for phenylketonuria (PKU) across the globe
Published in Metabolism Clinical and Experimental, this research article presents the real-world efficacy of PTC923, compared with sapropterin, in a Phase 2 study of patients with phenylketonuria (PKU)
This research article in Molecular Genetics and Metabolism Reports, reports results from a Phase 1 clinical study of CNSA-001 (sepiapterin), an oral treatment for BH4 deficiencies and phenylketonuria (PKU).
Published in Molecular Genetics and Metabolism Reports, this research article presents the results of a study investigating the potential effects of CNSA-001 – a preparation of sepiapterin – on neurometabolites in the central nervous system (CNS)
Always consult the prescribing information before prescribing any products
MED-US-CORP-2500008 | November 2025
MED-US-PKU-2600011 | February 2026