LMI 070 是一种高效、选择性和口服活性的小分子 SMN2 剪接调节剂，对 SMN 的EC50为 20 nM。它可提高全长 SMN 蛋白并延长其生存期，也抑制hERG，IC50为 6.3 μM。

LMI070 (NVS-SM1) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

To evaluate the efficacy of NVS-SM1, we used the SMNΔ7 mouse model, which displays a severe phenotype. In the specific colony used for this study, death typically occurs before postnatal day 15. We were pleased to observe that, in addition to a dose-dependent elevation of SMN protein in the brain, oral administration of NVS-SM1 improved body weight and extended lifespan, with 50% of the animals in the 1 mg per kg body weight group and 62% of animals in the 3 mg per kg body weight group showing increased survival.

1562338-42-4

C22H27N5O2

393.491

LMI 070;NVS-SM1

DMSO:6.12 mg/mL (15.55 mM)),Need ultrasonic and warming
Powder: -20°C for 3 years
In solvent: -80°C for 2 years