Home DNA Damage/DNA Repair SMN modulator Risdiplam

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Risdiplam SMN modulator

Cat.No.S6925

Risdiplam is an orally administered SMN2 pre-mRNA splicing modifier that distributes into the central nervous system (CNS) and peripheral tissues.This product has poor solubility, animal experiments are available, cell experiments please choose carefully!
Risdiplam SMN modulator Chemical Structure

Chemical Structure

Molecular Weight: 401.46

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Quality Control

Batch: S692501 DMSO]Insoluble]false]Water]Insoluble]false]Ethanol]Insoluble]false Purity: 99.58%
99.58

Solubility

In vitro
Batch:

DMSO : Insoluble
(Moisture-contaminated DMSO may reduce solubility. Use fresh, anhydrous DMSO.)

Water : Insoluble

Ethanol : Insoluble

Molarity Calculator

Mass Concentration Volume Molecular Weight
Dilution Calculator Molecular Weight Calculator

In vivo
Batch:

In vivo Formulation Calculator (Clear solution)

Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)

mg/kg
g
μL

Step 2: Enter the in vivo formulation (This is only the calculator, not formulation. Please contact us first if there is no in vivo formulation at the solubility Section.)

% DMSO
%
% Tween 80
% ddH2O
% DMSO
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%

Calculation results:

Working concentration: mg/ml;

Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )

Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.

Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.

Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such
as vortex, ultrasound or hot water bath can be used to aid dissolving.

Chemical Information, Storage & Stability

Molecular Weight 401.46 Formula

C22H23N7O

 Storage (From the date of receipt) 3 years -20°C powder
CAS No. 1825352-65-5 -- Storage of Stock Solutions

Read more about storage stability stock solution CAS number

Mechanism of Action

Targets/IC50/Ki
SMN2
References

Clinical Trial Information

(data from https://clinicaltrials.gov, updated on 2026-05-01)

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07047144 RECRUITING
Spinal Muscular Atrophy; SMA; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type 3; Neuromuscular Manifestations; Anti-myostatin
Scholar Rock, Inc.
 2025-09-15 PHASE2
NCT07444476 RECRUITING
Spinal Muscular Atrophy
Biogen
 2026-04-03 PHASE3
NCT05861986 RECRUITING
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2024-05-30 PHASE4
NCT05861999 RECRUITING
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2024-08-14 PHASE4
NCT05067790 ACTIVE_NOT_RECRUITING
Spinal Muscular Atrophy
Biogen
 2022-01-21 PHASE3
NCT05232929 ACTIVE_NOT_RECRUITING
Spinal Muscular Atrophy
Genentech, Inc.
 2022-03-29 PHASE4

Read more about Clinical Trials

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