Risdiplam

Product Description

Risdiplam is a selective survival of motor neuron-2 (SMN2) pre-mRNA splicing modifier. It potently enhances SMN2 splicing in vitro (EC_1.5X = 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.

Solubility

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 all affect the solvent volumes required to prepare stock solutions.

Select a batch to recalculate based on the batch molecular weight:

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Concentration / Solvent Volume / Mass	1 mg	5 mg	10 mg
1 mM	2.49 mL	12.45 mL	24.91 mL
5 mM	0.50 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	0.50 mL

Calculators

Background References

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

• Paik Risdiplam: A Review in Spinal Muscular Atrophy. CNS Drugs 2022 PMID: 35284988
• Martin Screening strategies for identifying RNA- and ribonucleoprotein-targeted compounds. Trends Pharmacol.Sci. 2021 PMID: 34215444
• Poirier Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol.Res.Perspect 2018 PMID: 30519476
• Zhao SMN protein is required throughout life to prevent spinal muscular atrophy disease progression. Hum.Mol.Genet. 2021 PMID: 34368854
• Markati Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA). Expert Opin.Investig.Drugs 2022 PMID: 35316106
• Ratni 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. 2018 PMID: 30044619