Show Notes
In this episode, Kyle Landskroner and Jagdeep S. Walia talk about their paper on nizubaglustat in a mouse model of GM2 gangliosidosis. They explore how this brain-penetrant dual GCS/NLGase inhibitor improved survival, motor function, and neuroinflammatory markers in Sandhoff disease mice, and what that could mean for future therapies in GM2 disease.
Therapeutic Effects of Nizubaglustat in a Mouse Model of GM2 Gangliosidosis Kyle Landskroner, Kshitiz Singh, Melissa Mitchell, Jagdeep S. Walia https://doi.org/10.1002/jimd.70130
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