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

View at publisher
Authors: Priya S. Kishnani, Mary‐Alice Abbott, Virginia E. Kimonis, Roberto Araujo, Julie L. Batista, Kelly George, Jennifer Heuterman, Kathryn M. Wilson, David W. Stockton
Cited by: 0

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
147
Issue
2
Pages
109502–109502
Publication date
1 February 2026
Language
EN