Work

Symptom onset, disease biomarkers, and treatment status in US Pompe disease patients identified by newborn screening

Year: 2026

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2025.109502

Authors: Priya S. Kishnani, Mary-Alice Abbott, Virginia Kimonis, Roberto Araujo, Julie L. Batista, Kelly George, Jennifer Heuterman, Kathryn M. Wilson, David W. Stockton

Institutions: Duke University, Baystate Medical Center, University of California, Irvine, Sanofi (Netherlands), Central Michigan University

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W7127397751

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