Risdiplam

Risdiplam is a survival of motor neuron-2 (SMN2) splicing modifier. It enhances SMN2 splicing (EC1.5x = 40 nM) and increases SMN protein levels (EC1.5x = 163 nM) in cellular assays. Risdiplam (3 mg/kg) increases brain and quadricep muscle SMN protein levels in the adult C/C-allele mouse model of mild spinal muscular atrophy (SMA) and the SMND7 neonate mouse model of severe SMA. It also increases the number of vesicular glutamate transporter 1 (vGLUT1) proprioceptive inputs on motor neurons, the number of L3 to L5 spinal motor neurons, the percentage of fully innervated neuromuscular junctions (NMJs), and the size of the extensor digitorum longus muscles in adult SMND7 mice. Formulations containing risdiplam have been used in the treatment of spinal muscular atrophy.Formal Name: 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. CAS Number: 1825352-65-5. Synonyms: RG-7916, RO7034067. Molecular Formula: C22H23N7O. Formula Weight: 401.5. Purity: >98%. Formulation: (Request formulation change), A crystalline solid. Solubility: Chloroform: 10 mg/ml. lambdamax: 255, 287, 404 nm. SMILES: O=C1C=C(C(C=C2C)=NN3C2=NC(C)=C3)N=C4N1C=C(N5CCNC6(CC6)C5)C=C4. InChi Code: 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: ASKZRYGFUPSJPN-UHFFFAOYSA-N.