Beneficial effects of acetyl-DL-Leucine (ADLL) in a mouse model of Sandhoff disease
Sandhoff disease is a rare neurodegenerative lysosomal storage disease associated with the storage of GM2 ganglioside in late endosomes/lysosomes. Here, we explored the efficacy of acetyl-DL-leucine (ADLL), which has been shown to improve ataxia in observational studies in patients with Niemann–Pick...
| Main Authors: | , , , , , |
|---|---|
| Format: | Journal article |
| Published: |
MDPI
2020
|