1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis
  3. Risdiplam

Risdiplam  (Synonyms: RG7916; RO7034067)

Cat. No.: HY-109101 Purity: 99.58%
COA Handling Instructions

Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels.

For research use only. We do not sell to patients.

Risdiplam Chemical Structure

Risdiplam Chemical Structure

CAS No. : 1825352-65-5

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Customer Review

Based on 6 publication(s) in Google Scholar

Other Forms of Risdiplam:

Top Publications Citing Use of Products
  • Biological Activity

  • Purity & Documentation

  • References

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Description

Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels[1].

IC50 & Target

SMN2

In Vitro

Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels. Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full length transcript and thus functional SMN protein. Spinal muscular atrophy (SMA) type I remains the most common genetic disease resulting in death in infancy. Characterized by progressive motor and respiratory muscle weakness, this autosomal recessive neuromuscular disorder is caused by low levels of the survival motor neuron protein (SMN) due to inactivating bi-allelic deletions and other disabling mutations in the survival motor neuron 1 (SMN1) gene[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Clinical Trial
Molecular Weight

401.46

Formula

C22H23N7O

CAS No.
Appearance

Solid

Color

White to yellow

SMILES

O=C(C=C(C(C=C1C)=NN2C1=NC(C)=C2)N=C3C=C4)N3C=C4N(CCN5)CC65CC6

Shipping

Room temperature in continental US; may vary elsewhere.

Storage
Powder -20°C 3 years
4°C 2 years
In solvent -80°C 6 months
-20°C 1 month
Solvent & Solubility
In Vitro: 

DMSO : 1.96 mg/mL (4.88 mM; ultrasonic and warming and adjust pH to 5 with HCl and heat to 60°C; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)

Ethanol : < 1 mg/mL (insoluble)

H2O : < 0.1 mg/mL (insoluble)

*Risdiplam is usually formulated as a suspension.

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 2.4909 mL 12.4545 mL 24.9091 mL
5 mM --- --- ---
View the Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

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Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

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Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

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In Vivo Dissolution Calculator
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Calculation results:
Working solution concentration: mg/mL
Purity & Documentation

Purity: 99.58%

References

Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

Optional Solvent Concentration Solvent Mass 1 mg 5 mg 10 mg 25 mg
DMSO 1 mM 2.4909 mL 12.4545 mL 24.9091 mL 62.2727 mL
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Risdiplam Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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Product Name:
Risdiplam
Cat. No.:
HY-109101
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