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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | The American College of Medical Genetics (ACMG) | ACMG 2025 poster: Interim results from the APHENITY extension study: Sepiapterin reduces blood Phe with improved dietary Phe tolerance in participants with phenylketonuria

ACMG 2025 poster: Interim results from the APHENITY extension study: Sepiapterin reduces blood Phe with improved dietary Phe tolerance in participants with phenylketonuria

This poster, presented at ACMG 2025, describes interim results from the ongoing APHENITY extension study, which evaluated the effect of sepiapterin on dietary Phe tolerance in a broad population of phenylketonuria (PKU) participants

  • Learn more about the updated APHENITY study results, including findings from the extension study of participants not involved in the original trial
  • Understand the long-term efficacy of sepiapterin on dietary Phe tolerance in participants of all ages and PKU disease severity
  • Review the safety profile of sepiapterin in this ongoing 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

This poster was developed and funded by PTC Therapeutics for ACMG 2025.

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MED-ALL-PKU-2500001 | October 2025

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