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Branaplam hydrochloride is an orally active, brain-permeable, selective modulator of SMN2 splicing, with an EC50 of 20 nM for SMN, and enhances full-length SMN protein and prolongs survival in a mouse model of severe spinal muscular atrophy (SMA). Branaplam hydrochloride also inhibits hERG with an IC50 of 6.3 μM.
| Molecular Weight | 429.94 |
| Formula | C22H28ClN5O2 |
| CAS Number | 1562338-39-9 |
| Form | Solid |
| Solubility (25°C) | DMSO >100 mg/mL Water >100 mg/mL |
| Storage | 4°C, protect from light, dry, sealed |
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