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Catalog Number | orb1908994 |
---|---|
Category | Small Molecules |
Description | Branaplam hydrochloride |
CAS Number | 1562338-39-9 |
MW | 429.95 |
SMILES | Cl.CC1(C)CC(CC(C)(C)N1)Oc1ccc(nn1)-c1ccc(cc1O)-c1cn[nH]c1 |
Formula | C22H28ClN5O2 |
Biological Activity | Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM). branaplam hydrochloride 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. |
Storage | Store at -20°C for 12 months. |
Note | For research use only |
Expiration Date | 12 months from date of receipt. |
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