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