Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics.

Objective: To present a case series that illustrates real-world use of pegvaliase based on the initial experiences of US healthcare providers.

Methods: Sixteen healthcare providers from 14 centers across the US with substantial clinical experience in treating patients with phenylketonuria (PKU) with pegvaliase in the two-plus years since FDA approval (May 2018) provided cases that exemplified important lessons from their initial experiences treating patients with pegvaliase. Key lessons from each case and takeaway points were discussed in both live and virtual meetings.