MPS I missense variants that cause a gain of glycosylation effect are amenable to treatment with novel glycosylation inhibitors
Fields of Study & Subjects
Lysosomal Storage Disorders Research
Glycogen Storage Diseases and Myoclonus
Glycosylation and Glycoproteins Research
Publication details
- Journal / source
- Molecular Genetics and Metabolism
- Volume
- 147
- Issue
- 2
- Pages
- 109626–109626
- Publication date
- 1 February 2026
- Language
- EN