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Home | Metabolism | Phenylketonuria (PKU) | Publications | Publication: Sepiapterin as a treatment for people living with phenylketonuria: A plain language summary of the APHENITY trial

Publication: Sepiapterin as a treatment for people living with phenylketonuria: A plain language summary of the APHENITY trial

This plain language summary of the APHENITY trial explains the long-term treatment with sepiapterin in patients with phenylketonuria (PKU) in a clear, non‑technical way for people without scientific or medical training.

  • Access a plain language overview of the APHENITY trial, based on an article published in the Lancet in 2024
  • Learn more about the effectiveness of the long-term treatment of PKU with sepiapterin
  • Understand the safety of sepiapterin in this study
▼ Sepiapterin is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-PTC923-2500012 | September 2025

Muntau AC,Gallagher S, Lah M et al. J Comp Eff Res 2026; Jan;15(1):e250116.

Once registered, you will be able to view the plain language summary as a PDF.

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MED-ALL-PKU-2600076 | April 2026

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