Nizubaglustat is a high potency, small molecule inhibitor of GCS and NLGase that is currently in Phase 3 trials for the treatment of Niemann-Pick type C (NPC) disease and GM1 and GM2 gangliosidoses. The dual mechanisms of action for nizubaglustat lends itself to potentially treat a variety of diseases in which there is lysosomal dysfunction or handling of glycolipids. Previously we have generated data in three genetically different mouse models of neurodegeneration that demonstrated increased survival and/or clinical benefits. The effects observed with nizubaglustat are likely due to its dual mode of action since specific inhibitors of each enzyme have not been effective in all three models. The underlying pathology shared across these diseases may also be shared by other neurologic disease such as CLN3 as the clinical phenotypes, e.g., ataxia, dysphasia, are shared across NPC, GM1 and GM2 gangliosidoses. The CLN3 protein is involved in endolysosomal trafficking and its loss leads to turnover of gangliosides. Moreover, mislocalization of gangliosides and autophagic flux have been linked to disease progression. Ganglioside dysregulation is detectable early and may play a role in CLN3 disease initiation. In this poster we will provide data on the potential application of nizubaglustat in rare neurodegenerative diseases.