Published in Genetics in Medicine, this review paper explores the current and future management and therapeutic strategies for phenylketonuria (PKU) across the globe
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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)
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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)
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MED-US-CORP-2500008 | November 2025
MED-US-PKU-2600011 | February 2026