1562338-39-9
1562338-39-9 structure
- Name: Branaplam hydrochloride
- Chemical Name: Branaplam hydrochloride
- CAS Number: 1562338-39-9
- Molecular Formula: C22H28ClN5O2
- Molecular Weight: 429.94
- Catalog: Signaling Pathways Cell Cycle/DNA Damage DNA/RNA Synthesis
- Create Date: 2022-08-09 15:24:47
- Modify Date: 2025-08-20 16:42:38
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Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model[1][2].
| Name | Branaplam hydrochloride |
|---|
| Description | Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model[1][2]. |
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| Related Catalog | |
| In Vitro | Branaplam (LMI070; NVS-SM1) hydrochloride treatment induces changes in the levels of 175 genes in human fibroblasts[1]. |
| In Vivo | Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) hydrochloride produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord[1]. Branaplam (1 mg/kg of IV; 3 mg/kg of PO) hydrochloride has a CL of 25 mL/min/kg and an AUC of 3.03 μM•h[2]. A single Branaplam (oral; 30 mg/kg) hydrochloride results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice[1]. Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) hydrochloride improves body weight and extendes lifespan in n SMNΔ7 mice[1]. |
| References |
| Molecular Formula | C22H28ClN5O2 |
|---|---|
| Molecular Weight | 429.94 |