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Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant inhibitor of glucosylceramide synthase (GCS) utilized in the investigation of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease.
Description | Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant inhibitor of glucosylceramide synthase (GCS) utilized in the investigation of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease. |
In vitro | Ibiglustat succinate (SAR402671) administered at 1 μM for 15 days to Fabry disease (FD) cells results in GL-3 levels nearly equivalent to those in untreated wild-type (WT) cells, indicating its potential to halt further GL-3 accumulation and mitigate excessive sphingolipid levels in FD cardiomyocytes[4]. |
Alias | Venglustat succinate, SAR402671succinate, Ibiglustat succinate, GZ402671succinate |
Molecular Weight | 507.58 |
Formula | C24H30FN3O6S |
Cas No. | 1629063-80-4 |
Relative Density. | no data available |
Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
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