Ibiglustat (L-Malic acid) is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. It can be 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. It can prevent additional GL-3 accumulation and could ameliorate the abundant levels of sphingolipid in FD cardiomyocytes.
- Orally active
- Brain-penetrant
- Glucosylceramide synthase (GCS) inhibitor
- Soluble in DMSO (≥ 100 mg/mL, 191.00 mM)
- Can be 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