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Risdiplam
本产品不向个人销售,仅用作科学研究,不用于任何人体实验及非科研性质的动物实验。
Risdiplam图片
CAS NO:1825352-65-5
包装与价格:
包装价格(元)
2 mg电议
5 mg电议
10 mg电议
25 mg电议
50 mg电议
100 mg电议
200 mg电议

产品名称
RG7916
RO7034067
产品介绍
Risdiplam 是一种中枢和外周分布的 SMN2 前 mRNA 剪接修饰剂,可增加运动神经元存活 (SMN) 蛋白水平。

产品描述

Risdiplam (RG7916) is orally administered. Risdiplam is a centrally and peripherally distributed SMN2 pre-mRNA splicing modifier which increases survival motor neuron (SMN) protein levels[1].

体外活性

Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full-length transcript and thus functional SMN protein. Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels [1]. Spinal muscular atrophy (SMA) type I remains the most common genetic disease resulting in death in infancy. Characterized by progressive motor and respiratory muscle weakness, this autosomal recessive neuromuscular disorder is caused by low levels of the survival motor neuron protein (SMN) due to inactivating bi-allelic deletions and other disabling mutations in the survival motor neuron 1 (SMN1) gene[1]. Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full-length transcript and thus functional SMN protein. Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels [1]. Spinal muscular atrophy (SMA) type I remains the most common genetic disease resulting in death in infancy. Characterized by progressive motor and respiratory muscle weakness, this autosomal recessive neuromuscular disorder is caused by low levels of the survival motor neuron protein (SMN) due to inactivating bi-allelic deletions and other disabling mutations in the survival motor neuron 1 (SMN1) gene[1].

Cas No.

1825352-65-5

分子式

C22H23N7O

分子量

401.46

别名

RG7916;RO7034067

储存和溶解度

Ethanol:2 mg/mL (4.98 mM),Need ultrasonic
DMSO:2 mg/mL (4.98 mM,Need ultrasonic
H2O:2.22 mg/mL (5.53 mM),ultrasonic and adjust pH to 6 with HCl
Powder: -20°C for 3 years
In solvent: -80°C for 2 years