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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | The Society for the Study of Inborn Errors of Metabolism (SSIEM) | SSIEM 2026 poster: Efficacy and safety of sepiapterin in individuals with PKU and baseline phenylalanine levels equal to or more than 900 μmol/l

SSIEM 2026 poster: Efficacy and safety of sepiapterin in individuals with PKU and baseline phenylalanine levels equal to or more than 900 μmol/l

This poster, presented at SSIEM 2026, examines the efficacy and safety of sepiapterin in individuals with phenylketonuria (PKU) and baseline phenylalanine (Phe) levels ≥900 µmol/L, including its impact on blood Phe levels and dietary protein tolerance

  • Review sepiapterin response rates in participants with high baseline Phe levels in three key studies
  • Gain insights into the proportion of participants achieving clinically meaningful reductions in blood Phe
  • Understand the safety profile of sepiapterin, and the potential for greater dietary flexibility for people living with PKU
▼ 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

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

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MED-ALL-PKU-2600171 | September 2026

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