CAS 1825352-65-5
:Risdiplam
Description:
Risdiplam is a small molecule drug primarily developed for the treatment of spinal muscular atrophy (SMA), a genetic disorder characterized by the loss of motor neurons. It functions as a survival motor neuron 2 (SMN2) splicing modifier, enhancing the production of functional SMN protein, which is crucial for motor neuron health. Risdiplam is characterized by its oral bioavailability, allowing for convenient administration compared to other therapies that require injections. The compound has a relatively low molecular weight and exhibits good solubility, which contributes to its pharmacokinetic profile. Its mechanism of action involves modulating the splicing of SMN2 pre-mRNA, thereby increasing the levels of SMN protein in the body. Risdiplam has undergone extensive clinical trials, demonstrating efficacy in improving motor function in patients with SMA. As with any medication, it is essential to monitor for potential side effects, which may include gastrointestinal disturbances and other systemic effects. Overall, Risdiplam represents a significant advancement in the therapeutic landscape for SMA.
Formula:C22H23N7O
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
InChI key:InChIKey=ASKZRYGFUPSJPN-UHFFFAOYSA-N
SMILES:O=C1N2C=C(N3CC4(CC4)NCC3)C=CC2=NC(=C1)C5=NN6C(C(C)=C5)=NC(C)=C6
Synonyms:- 7-(4,7-Diazaspiro[2.5]oct-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-4H-pyrido[1,2-a]pyrimidin-4-one
- Risdiplam
- RG 7916
- 4H-Pyrido[1,2-a]pyrimidin-4-one, 7-(4,7-diazaspiro[2.5]oct-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-
- RO 7034067
- RO7034067
- 7-amino-2,3-dihydro-1H-inden-4-ol
- RG7916
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Found 8 products.
2-(2,8-Dimethylimidazo[1,2-B]Pyridazin-6-Yl)-7-(4,7-Diazaspiro[2.5]Octan-7-Yl)-4H-Pyrido[1,2-a]Pyrimidin-4-One
CAS:2-(2,8-Dimethylimidazo[1,2-B]Pyridazin-6-Yl)-7-(4,7-Diazaspiro[2.5]Octan-7-Yl)-4H-Pyrido[1,2-a]Pyrimidin-4-OnePurity:99%Molecular weight:401.46g/molRisdiplam
CAS:Risdiplam (RG7916) is a centrally and peripherally distributed and orally administrable small molecule SMN2 pre-mRNA splicing modifier.Cost-effective and quality-assured.Formula:C22H23N7OPurity:98.68% - 99.64%Color and Shape:SolidMolecular weight:401.46Risdiplam
CAS:<p>Modifier of the SMN2 gene splicing</p>Formula:C22H23N7OPurity:Min. 98 Area-%Color and Shape:PowderMolecular weight:401.46 g/mol





