Nizubaglustat in GM2 Gangliosidosis

Nizubaglustat in GM2 Gangliosidosis

From JIMD Podcasts by Journal of Inherited Metabolic Disease

April 28, 2026 · 23 min

About this episode

Kyle Landskroner and Jagdeep S. Walia discuss the effects of nizubaglustat in a mouse model of GM2 gangliosidosis.

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

People in this episode

Guests: Kyle Landskroner, Jagdeep S. Walia

Topics covered

  • GM2 Gangliosidosis
  • Nizubaglustat
  • neuroinflammation
  • motor function
  • therapeutic effects

Keywords

  • nizubaglustat
  • GM2 gangliosidosis
  • Sandhoff disease
  • motor function
  • neuroinflammatory markers

Mentioned in this episode

Organizations: Journal of Inherited Metabolic Disease

Books & works: Therapeutic Effects of Nizubaglustat in a Mouse Model of GM2 Gangliosidosis

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