Ibiglustat (Venglustat) can cross the blood-brain barrier. Ibiglustat can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes.
|Solubility (25°C)||DMSO ≥ 40 mg/mL|
Powder -20°C 3 years ; 4°C 2 years
In solvent -80°C 6 months ; -20°C 1 month
|Body Surface Area (m2)||0.007||0.025||0.15||0.05||0.02||0.5|
|Animal A (mg/kg) = Animal B (mg/kg) multiplied by||Animal B Km|
|Animal A Km|
For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.
 Iva Stojkovska, et al. Cell Tissue Res. Molecular mechanisms of α-synuclein and GBA1 in Parkinson's disease
 Jean-Michel Itier, et al. J Inherit Metab Dis. Effective clearance of GL-3 in a human iPSC-derived cardiomyocyte model of Fabry disease
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