Sepiapterin Enables Liberalisation of Natural Protein Intake While Improving Metabolic Control in Adolescents with Phenylketonuria

Background

Sepiapterin is a synthetic precursor of tetrahydrobiopterin (BH4) that increases intracellular BH4 availability and has demonstrated greater phenylalanine-lowering efficacy than sapropterin. We evaluated its effect on dietary liberalisation and metabolic control in adolescents with phenylketonuria (PKU).