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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
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MED-US-CORP-2500008 | November 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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