Long-term outcomes of very early treated infantile-onset Pompe disease with the improvement after 24-month switching to avalglucosidase alfa: Real-world experiences based on Taiwan nationwide newborn screening program

Molecular Genetics and Metabolism 2026

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Authors: Chia-Feng Yang, Dau-Ming Niu

DOI: 10.1016/j.ymgme.2025.109681

Cited by: 0

Fields of Study & Subjects

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