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Tocris Bioscience™ Risdiplam
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Catalog No. 791650
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79-165-0 50 mg
79-161-0 10 mg
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Catalog No. 79-165-0 Supplier Tocris Bioscience™ Supplier No. 7916/50
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Description

Selective SMN2 gene splicing modifier

Risdiplam is a selective survival of motor neuron-2 (SMN2) pre-mRNA splicing modifier. It potently enhances SMN2 splicing in vitro (EC1.5X = 4 nM) and increases SMN protein in both brain (up to 100%) and muscle tissues (up to 49%) of transgenic mouse models of spinal muscular atrophy (SMA). Risdiplam dose-dependently increases the number of vesicular glutamate transporter 1 (vGlut1) proprioceptive inputs on motor neurons, the number of L3 to L5 motor neurons, the percentage of fully innervated neuromuscular junctions, and muscle size in the extensor digitorum longus muscles in SMN Δ7 mice.

Specifications

Chemical Name or Material 7-(4,7-Diazaspiro[2.5 ]oct-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-4 H-pyrido[1,2-a]pyrimidin-4-one
CAS 1825352-65-5
Quantity 50 mg
Target DNA, RNA and Protein Synthesis Compounds
Molecular Formula C22H23N7O
Purity 0.98
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