For research use only. Not for therapeutic Use.
Risdiplam (Cat No.:I020114) is a small molecule splicing modifier and selective survival of motor neuron-2 (SMN2) gene splicing modifier. It is an orally administered medication developed for the treatment of spinal muscular atrophy (SMA), a genetic neuromuscular disorder. Risdiplam increases the production of functional survival motor neuron (SMN) protein by promoting the inclusion of exon 7 during SMN2 pre-mRNA splicing. By increasing SMN protein levels, Risdiplam helps to improve motor neuron function and has shown significant clinical benefits in SMA patients, including improved muscle strength and motor function.
| Catalog Number | I020114 |
| CAS Number | 1825352-65-5 |
| Molecular Formula | C₂₂H₂₃N₇O |
| Purity | 98% |
| Target | DNA/RNA Synthesis |
| Target Protein | |
| Appearance | Solid |
| Storage | Dry, dark and at 2 - 8 °C for six months or -20°C for two years. |
| IUPAC Name | 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one |
| InChI | InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3 |
| InChIKey | ASKZRYGFUPSJPN-UHFFFAOYSA-N |
| SMILES | CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6 |
| Reference | [1]. Poirier A, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447. |
