Ibiglustat (Venglustat) L-Malic acid is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. It is utilized in the research of various conditions such as Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease. Studies suggest it can prevent additional GL-3 accumulation and ameliorate sphingolipid levels in conditions like Fabry disease.
- Orally active, brain-penetrant GCS inhibitor
- Used in research for Gaucher disease type 3
- Used in research for Parkinson's disease with GBA mutations
- Used in research for Fabry disease
- Used in research for GM2 gangliosidosis
- Used in research for autosomal dominant polycystic kidney disease
- Prevents GL-3 accumulation
- Ameliorates sphingolipid levels