Rewriting the natural history of late onset Pompe disease: A case series comparing patients diagnosed before and after newborn screening

Molecular Genetics and Metabolism 2026

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Authors: KeriAnn W. Kuperman, Christina Lee Grant

DOI: 10.1016/j.ymgme.2025.109509

Cited by: 0

Fields of Study & Subjects

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