A benefit-risk analysis of pegvaliase for the treatment of phenylketonuria: A study of patients' preferences

Abstract
Phenylketonuria (PKU) leads to an accumulation of phenylalanine (Phe) in the blood and subsequent neurologic, cognitive, psychiatric, and behavioral dysfunction. Many patients report social isolation and decreased quality of life. Pegvaliase is an enzyme substitution therapy that reduces blood Phe levels in patients with PKU and is associated with a risk of hypersensitivity reactions.To define the minimum acceptable benefit (MAB) of pegvaliase,...
Paper Details
Title
A benefit-risk analysis of pegvaliase for the treatment of phenylketonuria: A study of patients' preferences
Published Date
Dec 1, 2019
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