A structured methodology for evaluating patient-reported outcomes across a treatment program: A comparative application in migalastat

Molecular Genetics and Metabolism 2026

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Authors: Vera Gielen, Hoda Fotovvat, Siddharth Kakked, Biliana O. Veleva-Rotse, Christopher J. Wingrove, Elizabeth Nicki Bush

DOI: 10.1016/j.ymgme.2025.109425

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Health Systems Economic Evaluations Quality of Life Metabolism and Genetic Disorders