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Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM) and inhibits hERG (IC50: 6.3 μM). In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride increased full-length SMN protein and improved survival.
Pack Size | Price | Availability | Quantity |
---|---|---|---|
2 mg | $53 | 5 days |
Description | Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM) and inhibits hERG (IC50: 6.3 μM). In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride increased full-length SMN protein and improved survival. |
Molecular Weight | 429.95 |
Formula | C22H28ClN5O2 |
Cas No. | 1562338-39-9 |
Storage | Shipping with blue ice. |
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