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SMN-C3
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。
SMN-C3图片
CAS NO:1449597-34-5
包装与价格:
包装价格(元)
10 mg电议
50 mg电议
100 mg电议

产品介绍

产品描述

SMN-C3 is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA).

体内活性

At P16, the vehicle-treated D7 mice were much smaller than the control group of heterozygous litter pups and were dying. In contrast, D7 mice treated with high doses of SMN-C3 showed a phenotype similar to the heterozygous control.In the D7 mice, SMN-C3 treatment induces a dose-dependent bodyweight gain , with some animals showing a body weight that is ~80% that of heterozygous controls. SMN-C3 normalizes the motor behavior of D7 mice, illustrated by the ability of the mice to right themselves as quickly as heterozygous controls and by their level of locomotor activity. Most importantly, whereas vehicle-treated mice die within 3 weeks after birth with a median survival of 18 days, SMN-C3 treatment increases survival in a dose-dependent manner to a median survival time of 28 days in the low-dose (0.3 mg/kg per day) group. In the two higher-dose groups (1 and 3 mg/kg per day), ~90% of animals survive beyond P65 when the study is completed.

Cas No.

1449597-34-5

分子式

C24H28N6O

分子量

416.52

储存和溶解度

DMSO:5 mg/mL (12.00 mM),Need ultrasonic
Powder: -20°C for 3 years
In solvent: -80°C for 2 years