Home | Metabolism | Phenylketonuria (PKU) | Publications | Publication: Exploratory study of the effect of 1 week of orally-administered CNSA-001 (sepiapterin) on central nervous system (CNS) levels of tetrahydrobiopterin, dihydrobiopterin, and monoamine neurotransmitter metabolites in healthy volunteers
Publication: Exploratory study of the effect of 1 week of orally-administered CNSA-001 (sepiapterin) on central nervous system (CNS) levels of tetrahydrobiopterin, dihydrobiopterin, and monoamine neurotransmitter metabolites in healthy volunteers
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 neurometabolitesin the CNS
Results from a Phase 1 exploratory study are presented in this publication. Concentrations of molecules involved in neurometabolic pathways were recorded at baseline and day 7 in 8 healthy subjects who were administered oral CNSA-001 (n=6) or placebo (n=2) over 7 days
Levels of metabolites of interest measured in cerebrospinal fluid and plasma in this study – BH4, BH2, homovanillic acid, and 5-hydroxyindoleacetic acid (5-HIAA) – are presented
Smith N, Longo N, Levert K, et al.Mol Genet Metab Rep. 2019;21:100500
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MED-ALL-PKU-2100026 | October 2021
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