Long-term safety and efficacy of pegunigalsidase alfa in patients with Fabry disease: Results from the phase III BRILLIANCE extension study

Molecular Genetics and Metabolism 2026

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Authors: John A. Bernat, Aleš Linhart, Özlem Göker-Alpan, Shoshi Tessler, Meng Wang, Giovanni Piotti, Joana Almeida, Derralynn A. Hughes

DOI: 10.1016/j.ymgme.2025.109335

Cited by: 0

Fields of Study & Subjects

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