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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | The American College of Medical Genetics (ACMG) | ACMG 2025 symposium: Unlocking potential: Innovative advances in PKU treatment

ACMG 2025 symposium: Unlocking potential: Innovative advances in PKU treatment

This symposium, presented at ACMG 2025, summarizes the APHENITY study which assessed the effectiveness of sepiapterin on dietary Phe in a broad population of phenylketonuria (PKU) patients, and discusses the need for innovative treatment approaches

  • Learn more about the existing unmet needs in PKU
  • Review the results of the APHENITY study, including detailed case studies
  • Understand the need for innovative treatment across different populations of PKU patients
▼ 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 symposium was developed and funded by PTC Therapeutics for ACMG 2025.

The information in the materials was correct at the time of the events.

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MED-ALL-PKU-2500001 | March 2026

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