| Bioactivity | Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS). Lucerastat has the potential for Fabry disease study[1][2]. |
| Invitro | Fabry patient-derived fibroblasts with the genotypes R301G (residual -GalA activity; 20%) R220X (<3%) and W162X (<1%). Cell Viability Assay[2]. Cell Line: |
| In Vivo | Lucerastat (1200 mg/kg/day food admix), a GCS inhibitor, reduces Gb3 in the absence of residual -GalA activity[2]. Animal Model: |
| Name | Lucerastat |
| CAS | 141206-42-0 |
| Formula | C10H21NO4 |
| Molar Mass | 219.28 |
| Appearance | Solid |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | -20°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture) |
| Reference | [1]. Sanne J van der Veen, et al. Developments in the Treatment of Fabry Disease. J Inherit Metab Dis. 2020 Feb 21. [2]. R.W.D. Welford, et al. Lucerastat, an Iminosugar for Substrate Reduction Therapy in Fabry Disease: Preclinical Evidence. |
Lucerastat
CAS: 141206-42-0 F: C10H21NO4 W: 219.28
Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS). Lucera
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