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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | The Society for the Study of Inborn Errors of Metabolism (SSIEM) | SSIEM 2026 poster: EPIPHENY study design: Phase 3b study of long-term neurocognitive outcomes in sepiapterin-treated children with PKU

SSIEM 2026 poster: EPIPHENY study design: Phase 3b study of long-term neurocognitive outcomes in sepiapterin-treated children with PKU

This poster, presented at SSIEM 2026, outlines the design of the EPIPHENY Phase 3b study, which evaluates the effects of long-term sepiapterin on neurocognitive function in children with phenylketonuria (PKU)

  • Understand the study design and key inclusion and exclusion criteria of the ongoing EPIPHENY study
  • Assess how primary, secondary, and exploratory endpoints will be used to evaluate the efficacy and safety of long-term sepiapterin treatment, including effects on neurocognitive function and health-related quality of life
  • Review the demographics and clinical characteristics of participants enrolled to date
Always consult the prescribing information before prescribing any products

MED-US-CORP-2500008 | November 2025

​​This poster was developed and funded by PTC Therapeutics for SSIEM 2026.​

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