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• 产品描述： Branaplam (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 inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model

• 靶点： IC50: 20 nM (SMN) EC50: 6.3 μM (hERG);thers; PotassiumChannel; DNA/RNASynthesis

• 体外研究：
  Branaplam (LMI070; NVS-SM1) treatment induces changes in the levels of 175 genes in human fibroblasts

• 体内研究：
  Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord. Branaplam (1 mg/kg of IV; 3 mg/kg of PO) has a CL of 25 mL/min/kg and an AUC of 3.03 μM•h.A single Branaplam (oral; 30 mg/kg) results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice. Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) improves body weight and extendes lifespan in n SMNΔ7 mice. Animal Model: C/+ SMA mouse model Dosage: 3, 10, 30 mg/kg Administration: Oral Result: Produced dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord. Animal Model: Male Sprague-Dawley rat Dosage: 1 mg/kg (IV);3 mg/kg (PO) (Pharmacokinetic Analysis) Administration: IV or PO Result: Had a CL of 25 mL/min/kg and an AUC of 3.03 μM•h.

• 参考文献：
  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. 2. Cheung AK, et al. Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Dec 27;61(24):11021-11036.

• 溶解性： Soluble  in  DMSO

• 保存条件： -20℃

• 配置溶液浓度参考：
  

  	1mg	5mg	10mg
  1 mM	2.541 ml	12.707 ml	25.414 ml
  5 mM	0.508 ml	2.541 ml	5.083 ml
  10 mM	0.254 ml	1.271 ml	2.541 ml
  50 mM	0.051 ml	0.254 ml	0.508 ml

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本计算器可帮助您计算出特定溶液中溶质的质量、溶液浓度和体积之间的关系，公式为：

质量 (mg) = 浓度 (mM) x 体积 (mL) x 分子摩尔量 (g/mol)

• pg ng μg mg g kg =
  fM pM nM μM mM M *
  nL μL mL L *