Early disease manifestations and treatment outcomes in pediatric Fabry disease patients treated with agalsidase beta in the United States

Molecular Genetics and Metabolism 2026

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Authors: Dawn A. Laney, Roberto Araujo, Julie L. Batista, Kathryn M. Wilson, Kenneth I. Berger, Robert James Hopkin

DOI: 10.1016/j.ymgme.2025.109513

Cited by: 0

Fields of Study & Subjects

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