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Home | Metabolism | Phenylketonuria (PKU) | Publications | Publication: Global considerations for lifelong management and therapeutic development for phenylketonuria

Publication: Global considerations for lifelong management and therapeutic development for phenylketonuria

Published in Genetics in Medicine, this review paper explores the current and future management and therapeutic strategies for phenylketonuria (PKU) across the globe

  • Learn more about PKU, a rare metabolic disorder
  • Review the current management strategies for PKU used in different countries, from China to Italy
  • Understand the data surrounding the role of sepiapterin in PKU and its clinical implications
▼ 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

Longo N, Hamazaki T, Hollander S, et al. Genet Med. Published online August 5, 2025.

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MED-ALL-PKU-2500177 | October 2025

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