Shopping Cart
- Remove All
- Your shopping cart is currently empty
Branaplam hydrochloride (LMI070; NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 μM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival.
Pack Size | Price | Availability | Quantity |
---|---|---|---|
1 mg | $39 | In Stock | |
5 mg | $83 | In Stock | |
10 mg | $118 | In Stock | |
25 mg | $198 | In Stock | |
50 mg | $293 | In Stock | |
100 mg | $433 | In Stock | |
200 mg | $642 | In Stock | |
1 mL x 10 mM (in DMSO) | $98 | In Stock |
Description | Branaplam hydrochloride (LMI070; NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 μM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival. |
Targets&IC50 | SMN:20 nM (EC50), ERG (human):6.3 μM |
In vitro | In Homo sapiens fibroblasts, Branaplam hydrochloride caused changes in the expression levels of 175 genes. [1] |
In vivo | After oral administration of Branaplam hydrochloride (3, 10, 30 mg/kg), the SMN2-FL transcript and SMN protein levels in the brain and spinal cord of mice showed a dose-dependent increase [1]. |
Synonyms | NVS-SM1 hydrochloride, LMI070 hydrochloride, LMI 070 hydrochloride |
Molecular Weight | 429.95 |
Formula | C22H28ClN5O2 |
Cas No. | 1562338-39-9 |
Smiles | Cl.OC1=CC(=CC=C1C=2N=NC(OC3CC(NC(C)(C)C3)(C)C)=CC2)C=4C=NNC4 |
Color | Yellow |
Appearance | Solid |
Storage | keep away from moisture,keep away from direct sunlight | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. | |||||||||||||||||||||||||||||||||||
Solubility Information | H2O: <1mg/mL (Insoluble) ![]() DMSO: 200 mg/mL (465.17 mM), Sonication is recommended. ![]() | |||||||||||||||||||||||||||||||||||
Solution Preparation Table | ||||||||||||||||||||||||||||||||||||
DMSO
|
Copyright © 2015-2025 TargetMol Chemicals Inc. All Rights Reserved.