Use of Pegvaliase in the Management of Phenylketonuria: Case Series of Early Experience in US Clinics

    Darius J. Adams, Hans C. Andersson, Heather Bausell, Kea Crivelly, Caroline Eggerding, Melissa Lah, Joshua Lilienstein, Kristin Lindstrom, Markey C. McNutt, Joseph W. Ray, Heather Saavedra, Stephanie Sacharow, Danielle Starin, Jennifer Tiffany-Amaro, Janet A. Thomas, Erika Vucko, Leah B. Wessenberg, Kaleigh Bulloch Whitehall
    Studysummary This case series highlights the real-world challenges and experiences of US healthcare providers using pegvaliase for phenylketonuria treatment, emphasizing patient education on adverse events and dietary changes, and tailored titration schedules, while reporting that most patients achieved treatment efficacy despite initial adverse events.
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