Risdiplam

Catalog # Availability Size / Price Qty
7916/50
7916/10
Risdiplam | CAS No. 1825352-65-5 | DNA, RNA and Protein Synthesis Compounds
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Description: Selective SMN2 gene splicing modifier

Chemical 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

Purity: ≥98%

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Biological Activity

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.

Technical Data

M.Wt:
401.47
Formula:
C22H23N7O
Solubility:
Soluble to 100 mM in 1eq. HCl with gentle warming
Purity:
≥98%
Storage:
Store at -20°C
CAS No:
1825352-65-5

The technical data provided above is for guidance only. For batch specific data refer to the Certificate of Analysis.
Tocris products are intended for laboratory research use only, unless stated otherwise.

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