Lessons learned from 5 years of pegvaliase in US clinics: A case series.

ABSTRACT:

Objective

To provide insights and strategies for pegvaliase management in challenging cases with phenylketonuria (PKU) based on the first 5 years of experience with pegvaliase in real-world clinical practice.

Methods

Twelve PKU experts gathered during a one-day, in-person meeting to discuss clinical cases illustrating important lessons from their experiences treating patients with pegvaliase in real-world clinical practice. Challenges with pegvaliase experienced prior to and during treatment and corresponding strategies to overcome them were discussed.

Results

Twelve cases of adults with PKU (eight females and four males, aged 18 to 68 years) receiving pegvaliase were reviewed and discussed. Challenges of the cases included medical or mental health comorbidities, exec

SUBMITTER: Cooney E 

PROVIDER: S-EPMC11733187 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature