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

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

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 clinic...

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Main Authors: Erin Cooney, Zineb Ammous, Tricia Bender, Gillian E. Clague, Marilyn Clifford, Angela Crutcher, Laura Davis-Keppen, Kirsten Havens, Melissa Lah, Stephanie Sacharow, Amarilis Sanchez-Valle, Erika Vucko, Bridget Wardley, Leah Wessenberg, Hans C. Andersson
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Phenylketonuria
PKU
Pegvaliase
Case series
Expert opinion
Treatment
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426924001344
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