A transferrin receptor-targeted α-L-fucosidase, JR-471, reduced core-fucosylated glycoasparagine in the brain and preserved motor function in a murine model of fucosidosis
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
- 109544–109544
- Publication date
- 1 February 2026
- Language
- EN