Risdiplam
Based on 9 publication(s) in Google Scholar
Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels.
Para uso exclusivo en investigación. No vendemos a pacientes.
- Pureza: 99.64%
- No. CAS: 1825352-65-5
- Fòrmula: C22H23N7O
- Peso molecular:401.46
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Almacenamiento:Powder -20°C, 3 years , 4°C, 2 years ; In solvent -80°C, 6 months , -20°C, 1 month
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Actividad biológica
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Chemical Information
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Solvente y solubilidad
- Pureza y Documentación
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Publications Citing Use of MedChemExpress (MCE) Risdiplam
More- Nat Commun. 2023 Jun 10;14(1):3435. [Abstract]
- Nucleic Acids Res. 2023 Jul 7;51(12):5948-5980. [Abstract]
- Nucleic Acids Res. 2021 Sep 7;49(15):8462-8470. [Abstract]
- Proc Natl Acad Sci U S A. 2026 Mar 31;123(13):e2525423123. [Abstract]
- Cell Mol Life Sci. 2022 Jul 22;79(8):441. [Abstract]
- RSC Adv. 2026 May 19;16(29):27057-27068. [Abstract]
- Neurosci Insights. 2024 Feb 19:19:26331055241233596. [Abstract]
- Res Sq. 2026 Jun 11.
- bioRxiv. 2023 Jul 27:2023.07.25.550489. [Abstract]
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Actividad biológica
SMN2
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.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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No. CAS 1825352-65-5
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Appearance Solid
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Peso molecular 401.46
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Fòrmula C22H23N7O
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Color White to yellow
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SMILES
O=C(C=C(C(C=C1C)=NN2C1=NC(C)=C2)N=C3C=C4)N3C=C4N(CCN5)CC65CC6
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Synonyms
RG7916; RO7034067
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month
Publications (9)
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Journal Impact Factor
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Most Recent
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Nat Commun
2023 Jun 10;14(1):3435. PMID: 37301863
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.
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Nucleic Acids Res
Diverse targets of SMN2-directed splicing-modulating small molecule therapeutics for spinal muscular atrophy. [Abstract]2023 Jul 7;51(12):5948-5980. PMID: 37026480 -
Nucleic Acids Res
A small-molecule fluorescence probe ANP77 for sensing RNA internal loop of C, U and A/CC motifs and their binding molecules. [Abstract]2021 Sep 7;49(15):8462-8470. PMID: 34358308 -
Proc Natl Acad Sci U S A
2026 Mar 31;123(13):e2525423123. PMID: 41886383
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.
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Cell Mol Life Sci
2022 Jul 22;79(8):441. PMID: 35864358 -
RSC Adv
An approach for the systematic profiling of drug-induced remodeling of RNA-RBP (RNA-binding protein) interactions. [Abstract]2026 May 19;16(29):27057-27068. PMID: 42170088 -
Neurosci Insights
Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene. [Abstract]2024 Feb 19:19:26331055241233596. PMID: 38379891 -
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bioRxiv
PMS1 as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington's disease. [Abstract]2023 Jul 27:2023.07.25.550489. PMID: 37547003
Solvente y solubilidad
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.
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.
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.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Pureza y Documentación
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Ficha de datos (283 KB)
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SDS (393 KB)
- English - EN (393 KB)
- Français - FR (393 KB)
- Deutsch - DE (393 KB)
- Norwegian - NO (393 KB)
- Español - ES (393 KB)
- Swedish - SV (393 KB)
- Italian - IT (393 KB)
- Korean - KR (393 KB)
- Portuguese - PT (393 KB)
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Instrucciones de manejo (2659 KB)
Referencias
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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