Real-world effectiveness of migalastat versus enzyme replacement therapy in previously treatment-naïve patients with Fabry disease: Analyses of matched populations from the global followME Pathfinders registry

Molecular Genetics and Metabolism 2026

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Authors: Peter Nordbeck, Agnieszka Kopiec, Noemi Hummel, Vera Gielen, Biliana O. Veleva-Rotse, Alasdair C. Macculloch

DOI: 10.1016/j.ymgme.2025.109565

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Metabolism and Genetic Disorders