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
The American College of Medical Genetics (ACMG)
The American College of Medical Genetics (ACMG) brings together medical genetics professionals and industry leaders to discuss the latest discoveries and best practice in genetic disorders, rare diseases, genome sequencing, gene therapies and genetic counseling.
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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
▼ 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
MED-ALL-PKU-2500224 | October 2025