This page tracks new and upcoming treatment options for GM1 gangliosidosis. What’s in development? What’s promising? What’s available soon? Current topics include LYS-GM101, gene therapy, Miglustat, N-alkylated sugar, and more. Jump to section: News, Research, Clinical Trials
Last updated: 4 years ago
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Research
- Mechanistic Insights into the Chaperoning of Human Lysosomal-Galactosidase Activity: Highly Functionalized Aminocyclopentanes and C -5a-Substituted Derivatives of 4-epi -Isofagomine Sep 9, 2020
- Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience Aug 12, 2020