A SMN2 splicing modifier
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Risdiplam

Item No. 29028

Product Insert (PDF)Safety Data Sheet (SDS) (PDF)
Technical Information
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(Learn about Variance in Solubility)
Chloroform: 10 mg/ml
λmax
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
Shipping & Storage Information
Storage
-20°C
Shipping
Room temperature in continental US; may vary elsewhere
Certificates of Analysis & Batch Specific Data

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    1 mg

    $41

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    5 mg

    $128

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    10 mg

    $235

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    25 mg

    $389

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    Product Description

    Risdiplam is a survival of motor neuron-2 (SMN2) splicing modifier.1 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.

    WARNING This product is not for human or veterinary use.

    References & Product Citations
    Product Description References

    1. Ratni, H., Ebeling, M., Baird, J., et al. Discovery of risdiplam, a selective survival of motor neuron-2 (SMN2) gene splicing modifier for the treatment of spinal muscular atrophy (SMA). J. Med. Chem. 61(15), 6501-6517 (2018).