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Risdiplam

Cat. No. 7916 提交评论 说明书 / 分析证书（CoA） / 化学品安全技术说明书（SDS）
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说明： Selective SMN2 gene splicing modifier
化学名： 7-(4,7-Diazaspiro[2.5]oct-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-4H-pyrido[1,2-a]pyrimidin-4-one
纯度： ≥98% (HPLC)
说明书
引用文献
评论
文献 (2)
生物活性 技术数据 溶解性 计算器 说明书 参考文献

生物活性 for Risdiplam

Risdiplam is a selective survival of motor neuron-2 (SMN2) pre-mRNA splicing modifier. It potently enhances SMN2 splicing in vitro (EC1.5X = 4 nM) and increases SMN protein in both brain (up to 100%) and muscle tissues (up to 49%) of transgenic mouse models of spinal muscular atrophy (SMA). Risdiplam dose-dependently increases the number of vesicular glutamate transporter 1 (vGlut1) proprioceptive inputs on motor neurons, the number of L3 to L5 motor neurons, the percentage of fully innervated neuromuscular junctions, and muscle size in the extensor digitorum longus muscles in SMN Δ7 mice.

技术数据 for Risdiplam

分子量 401.47
公式 C22H23N7O
储存 Store at -20°C
纯度 ≥98% (HPLC)
CAS Number 1825352-65-5
PubChem ID 118513932
InChI Key ASKZRYGFUPSJPN-UHFFFAOYSA-N
Smiles O=C1C=C(N=C2C=CC(=CN12)N3CCNC4(C3)CC4)C5=NN6C=C(N=C6C(=C5)C)C

上方提供的技术数据仅供参考。批次相关数据请参见分析证书。

Tocris products are intended for laboratory research use only, unless stated otherwise.

溶解性数据 for Risdiplam

溶剂 最高浓度 mg/mL 最高浓度 mM
溶解性
1eq. HCl 40.15 100 温和加热

制备储备液 for Risdiplam

以下数据基于产品分子量 401.47。 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

选择批次从而根据批次分子量重新计算:
浓度/溶剂体积/质量 1 mg 5 mg 10 mg
1 mM 2.49 mL 12.45 mL 24.91 mL
5 mM 0.5 mL 2.49 mL 4.98 mL
10 mM 0.25 mL 1.25 mL 2.49 mL
50 mM 0.05 mL 0.25 mL 0.5 mL

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参考文献 for Risdiplam

参考文献是支持产品生物活性的出版物。

Ratni et al (2018) Discovery of Risdiplam, a selective survival of motor neuron-2 (SMN2) gene splicing modifier. for the treatment of spinal muscular atrophy (SMA). J.Med.Chem. 61 6501 PMID: 30044619

Markati et al (2022) Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA). Expert Opin.Investig.Drugs 31 451 PMID: 35316106

Zhao et al (2021) SMN protein is required throughout life to prevent spinal muscular atrophy disease progression. Hum.Mol.Genet. 31 82 PMID: 34368854

Poirier et al (2018) Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol.Res.Perspect 6 e00447 PMID: 30519476

Martin et al (2021) Screening strategies for identifying RNA- and ribonucleoprotein-targeted compounds. Trends Pharmacol.Sci. 42 758 PMID: 34215444

Paik et al (2022) Risdiplam: A Review in Spinal Muscular Atrophy. CNS Drugs 36 401 PMID: 35284988

If you know of a relevant reference for Risdiplam, please let us know.

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关键词: Risdiplam, Risdiplam supplier, Risdiplam, RO7034067, RG7916, survival, of, motor, neuron-2, SMN2, pre-mRNA, splicing, modifier, modulator, spinal, muscular, atrophy, SMA, DNA,, RNA, and, Protein, Synthesis, 7916, Tocris Bioscience

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