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ML372 inhibits survival motor neuron (SMN) protein ubiquitination, increases SMN protein stability without affecting mRNA expression. ML372 improves spinal muscular atrophy (SMA) in mice. ML372 is brain penetrant and has a reasonable exposure and half-life in vivo.
| Molecular Weight | 360.43 |
| Formula | C18H20N2O4S |
| CAS Number | 1331745-61-9 |
| Form | Solid |
| Solubility (25°C) | DMSO 33.33 mg/mL (ultrasonic and warming and heat to 60°C) |
| Storage |
Powder -20°C 3 years ; 4°C 2 years In solvent -80°C 6 months ; -20°C 1 month |
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