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

Risdiplam  (Synonyms: RG7916; RO7034067)

Cat. No.: HY-109101 Purity: 99.64%
COA
SDS
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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.

CAS No. : 1825352-65-5

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Customer Review

Based on 7 publication(s) in Google Scholar

Other Forms of Risdiplam:

Risdiplam Related Antibodies

Top Publications Citing Use of Products

    Risdiplam purchased from MedChemExpress. Usage Cited in: Proc Natl Acad Sci U S A. 2026 Mar 31;123(13):e2525423123.  [Abstract]

    Representative Oil Red O (ORO) staining images and quantitative analysis of FAPs 5 days after induction. Smn1 knockdown increased the ORO area in control FAPs, while Risdiplam (1 μM) treatment significantly decreased the ORO area in SMA FAPs.

    Risdiplam purchased from MedChemExpress. Usage Cited in: Proc Natl Acad Sci U S A. 2026 Mar 31;123(13):e2525423123.  [Abstract]

    Immunoblot analysis was performed on PDGFRα, Perilipin, SMN, and GAPDH before and after induction, with or without 1 µM Risdiplam treatment.

    Risdiplam purchased from MedChemExpress. Usage Cited in: Proc Natl Acad Sci U S A. 2026 Mar 31;123(13):e2525423123.  [Abstract]

    RT-qPCR analysis was performed on RNA isolated from 0 dpi FAPs to detect key genes controlling adipocyte differentiation. Risdiplam (1 μM) treatment restored the expression of Pparg, Adipoq, and Cebpa genes in SMA FAPs to normal. Smn1 knockdown increased the expression of these target genes in control FAPs.

    Risdiplam purchased from MedChemExpress. Usage Cited in: Nat Commun. 2023 Jun 10;14(1):3435.  [Abstract]

    Linear regression of gene expression changes in SMN2 splicing reporter following Risdiplam treatment with different doses for stable lines under the regulation of different human promoters and promoter strength measured by basal RT-qPCR in control conditions.

    Risdiplam purchased from MedChemExpress. Usage Cited in: Nat Commun. 2023 Jun 10;14(1):3435.  [Abstract]

    RNA polymerase occupancy at the promoter region or gene body was evaluated by CUT&RUN-qPCR using a specific antibody (Rpb1 CTD) in HEK293T-A2 stable cell lines expressing the SMN2 splicing reporter and treated with Risdiplam 10 uM.

    View All DNA/RNA Synthesis Isoform Specific Products:

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    RNASE L DNA Polymerases RNA Polymerases Helicases DNA Ligase

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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.

    Risdiplam Related Antibodies
    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)

    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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    In Vivo Dissolution Calculator
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    Purity & Documentation

    Purity: 99.64%

    SDS (393 KB)

    COA (247 KB) HNMR (253 KB) LCMS (94 KB)

    Handling Instructions (2659 KB)
    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.

    Keywords:

    Risdiplam1825352-65-5RG7916 RO7034067RG 7916RG-7916RO7034067RO 7034067RO-7034067DNA/RNA SynthesisInhibitorinhibitorinhibit

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