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NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research.
| Molecular Weight | 406.52 |
| Formula | C23H30N6O |
| CAS Number | 1562333-92-9 |
| Form | Solid |
| Solubility (25°C) | DMSO 75 mg/mL (ultrasonic) |
| Storage | Powder -20°C |
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