Lucerastat (1200 mg/kg/day food admix), a GCS inhibitor, reduces Gb3 in the absence of residual-GalA activity[2].
| Animal Model: | Fabry mice (Gla-/0 and Gla-/-, n = 5 or 6 for each gender)[2]. |
| Dosage: | 1200 mg/kg/day food admix. |
| Administration: | Food admix for 20 weeks. |
| Result: | Reduced lipid storage in two major organs affected by FD: mean Gb3 in the kidneys (-33%, p<0.01). and α-galactose- terminated glycosphingolipids in the dorsal root ganglia (-48%, p<0.05). In the liver of the Fabry mice, mean glucosylceramide (GlcCer (24:0)) was reduced (-59%, p<0.001) in addition to Gb3 (24:1) (-37%, p<0.05) demonstrating substrate reduction through GCS inhibition. |
