Ibiglustat succinate is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. It is used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease.
- Orally active
- Brain-penetrant glucosylceramide synthase (GCS) inhibitor
- Can prevent additional GL-3 accumulation in Fabry disease cells