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Phenylketonuria (PKU)

Learn more about PKU and BioMarin therapies

PKU Clinical Program

Explore select publications on PKU and the clinical research on BioMarin therapies.

Video

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  • Educational Materials
  • Phenylketonuria (PKU)

PKU:
Burden of Illness

Dr. Deborah Bilder
University of Utah

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Flyer

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  • Educational Materials
  • Phenylketonuria (PKU)

Answers in CME: Effective Communication Strategies for Improved Phenylketonuria Care

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Resource

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  • Educational Materials
  • Phenylketonuria (PKU)
  • 2024

Statement regarding ex vivo PAL enzymatic activity at ambient temperature

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Selected Publications

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  • Educational Materials
  • Phenylketonuria (PKU)

Access selected publications on the burden of illness of PKU, sapropterin dihydrochloride, and pegvaliase.

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Resource

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  • Educational Materials
  • Phenylketonuria (PKU)

Neurocognitive, neurological and neuropsychological manifestations of Phenylketonuria (PKU)

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PKU Congress Posters and Presentations

Access posters and presentations from recent congresses

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