From frustration to function: Reimagining registry value with individual monitoring dashboards to visualize disease progression in Pompe disease

Molecular Genetics and Metabolism 2026

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Authors: Paul T. McIntosh, Brad Crittenden, Sophie Clarke, Vipul Jain, Kinesha O'Brien-Prince, Thomas Scherer, Stephan Wenninger

DOI: 10.1016/j.ymgme.2025.109545

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Biomedical Research and Pathophysiology