Bioactivity:

NVS-SM2 is an effective small molecule splicing enhancer of SMN2 with oral activity. NVS-SM2 showed increased exon 7 inclusion and upregulated SMN protein expression in SMA fibroblasts and SMN cells _7 5025 mouse myoblasts.

CAS nr:

1562333-92-9

Purity:

98%

Molecular Weight:

406,53

SMILES:

OC1=CC(C2=CNN=C2)=CC=C1C3=NN=C(N(C)C4CC(C)(C)NC(C)(C)C4)C=C3

Formula:

C23H30N6O

Pathway:

Target: