Risdiplam

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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% (HPLC)
Datasheet
Citations
Reviews
Literature (2)

Biological Activity for Risdiplam

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 for Risdiplam

M. Wt 401.47
Formula C22H23N7O
Storage Store at -20°C
Purity ≥98% (HPLC)
CAS Number 1825352-65-5
PubChem ID 118513932
InChI Key ASKZRYGFUPSJPN-UHFFFAOYSA-N
Smiles O=C1C=C(N=C2C=CC(=CN12)N3CCNC4(C3)CC4)C5=NN6C=C(N=C6C(=C5)C)C

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.

Solubility Data for Risdiplam

Solvent Max Conc. mg/mL Max Conc. mM
Solubility
1eq. HCl 40.15 100 with gentle warming

Preparing Stock Solutions for Risdiplam

The following data is based on the product molecular weight 401.47. Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Select a batch to recalculate based on the batch molecular weight:
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 2.49 mL 12.45 mL 24.91 mL
5 mM 0.5 mL 2.49 mL 4.98 mL
10 mM 0.25 mL 1.25 mL 2.49 mL
50 mM 0.05 mL 0.25 mL 0.5 mL

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References for Risdiplam

References are publications that support the biological activity of the product.

Ratni et al (2018) 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 6501 PMID: 30044619

Markati et al (2022) Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA). Expert Opin.Investig.Drugs 31 451 PMID: 35316106

Zhao et al (2021) SMN protein is required throughout life to prevent spinal muscular atrophy disease progression. Hum.Mol.Genet. 31 82 PMID: 34368854

Poirier et al (2018) Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol.Res.Perspect 6 e00447 PMID: 30519476

Martin et al (2021) Screening strategies for identifying RNA- and ribonucleoprotein-targeted compounds. Trends Pharmacol.Sci. 42 758 PMID: 34215444

Paik et al (2022) Risdiplam: A Review in Spinal Muscular Atrophy. CNS Drugs 36 401 PMID: 35284988


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Keywords: Risdiplam, Risdiplam supplier, Risdiplam, RO7034067, RG7916, survival, of, motor, neuron-2, SMN2, pre-mRNA, splicing, modifier, modulator, spinal, muscular, atrophy, SMA, DNA,, RNA, and, Protein, Synthesis, 7916, Tocris Bioscience

Citations for Risdiplam

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Literature in this Area

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