Early enzyme replacement therapy in late-onset Pompe disease diagnosed by newborn screening

Molecular Genetics and Metabolism 2026

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Authors: Laura E. Case, Erin Huggins, Harrison N. Jones, Lisa D. Hobson‐Webb, Deborah R. McMechan, Neelam A. Makhijani, Erica Burner Nading, Steven A. Moore, Priya S. Kishnani
DOI: 10.1016/j.ymgme.2026.109864
Cited by: 0
References: 28

Fields of Study & Subjects

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

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
148
Issue
2
Pages
109864–109864
Publication date
4 March 2026
Language
EN