Branaplam (LMI070) (Synonyms: Branaplam, NVS-SM1) |
| Catalog No.GC32702 |
Branaplam (LMI070) (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 (LMI070) inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam (LMI070) elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.
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Cas No.: 1562338-42-4
Sample solution is provided at 25 µL, 10mM.
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Branaplam (LMI070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.
[1]. Palacino J, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-517.
Average Rating: 5 (Based on Reviews and 11 reference(s) in Google Scholar.)
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