Work

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

Year: 2026

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2025.109357

Authors: Laura E. Case, Erin Huggins, Harrison N. Jones, Lisa D. Hobson-Webb, Deborah R. McMechan, Neelam A. Makhijani, Erica Nading, Steven A. Moore, Priya S. Kishnani

Institutions: Duke University, East Carolina University, Wake Forest University, University of Iowa

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

Funders: Amicus Therapeutics, Sanofi Genzyme

OpenAlex ID: W7127274039

FWCI:

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