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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | Society for Inherited Metabolic Disorders (SIMD) | SIMD 2024 symposium: Advancing care for PKU: Results from the Phase 3 APHENITY trial

SIMD 2024 symposium: Advancing care for PKU: Results from the Phase 3 APHENITY trial

This symposium, presented at SIMD 2024, discusses the results of the Phase 3 APHENITY trial, which investigated the safety and efficacy of sepiapterin in patients with phenylketonuria (PKU)

  • Understand the remaining unmet needs in PKU and potential for improvements in treatment and care
  • Learn about oral sepiapterin, and its distinct mechanism of action
  • Review the results of the APHENITY Phase 3, global, multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating oral sepiapterin in a broad range of participants with PKU
  • Evaluate the APHENITY open-label extension study and its preliminary results, including findings from the dietary phenylalanine tolerance assessment
▼ 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 SIMD 2024.

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MED-ALL-PKU-2400086 | October 2025

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