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Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.
| Molecular Weight | 393.48 |
| Formula | C22H27N5O2 |
| CAS Number | 1562338-42-4 |
| Solubility (25°C) | DMSO 4 mg/mL |
| Storage | 2-8°C, dry, sealed |
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