Risdiplam (RG7916) (Synonyms: RG-7916, RO7034067)

Name: Risdiplam (RG7916)
Brand: GlpBio
SKU: GC30845
Availability: InStock
Rating: 5 (21 reviews)

カタログ番号GC30845

Risdiplamは、SMN2 pre-mRNAのスプライシング修飾剤であり、エクソン7の含有と完全な長さのSMN2 mRNAの生成を促進する。これにより、SMN1の欠損を補償することができる。

Products are for research use only. Not for human use. We do not sell to patients.

Risdiplam (RG7916) 化学構造

Cas No.: 1825352-65-5

サイズ

価格

在庫数

個数

5mg	$105.00	在庫あり
10mg	$175.00	在庫あり
25mg	$315.00	在庫あり
50mg	$595.00	在庫あり
100mg	$945.00	在庫あり

Tel：(909) 407-4943 Email: sales@glpbio.com

顧客レビュー

カスタマーレビューに基づきます。

Sample solution is provided at 25 µL, 10mM.

評判の良い論文で引用されたGlpBio製品

Nature
610.7931 (2022): 366-372

Nature
618, 1017–1023 (2023)

Cell
183.7 (2020): 1867-1883

Cell Research
31, 1291–1307 (2021)

Cell Research
33, 273–287 (2023)

Cell Research
33, 546–561 (2023)

Molecular Cancer
21.1 (2022): 1-17

Molecular Cancer
21.1 (2022): 1-18

Cancer Cell
39 (2021): 1-16

Cell Host Microbe
30.8 (2022): 1124-1138

Cell Host Microbe
31.5 (2023): 766-780

Cell Host Microbe
31.3 (2023): 418-43

Cell Metabolism
34.2 (2022): 240-255

Ann Rheum Dis
82(4): 533-545 (2023)

Cell Mol Immunol
20, 264–276 (2023)

Adv Funct Mater
33.35 (2023): 2214998

Description Protocol Chemical Properties Product Documents

Product Documents

Quality Control & SDS

View current batch:

Purity: >99.00%

Protocol

Cell experiment [1]:
Cell lines	SMA type I fibroblasts; SMA type I motor neurons
Reaction Conditions	0-1µM
Applications	Risdiplam (RG7916) was active in vitro in SMA patient-derived fibroblasts and in motor neurons generated from induced pluripotent stem cells (iPSCs) derived from SMA type 1 patient fibroblasts, promoting the inclusion of exon 7, to generate full-length (FL) mRNA.
Animal experiment [2]:
Animal models	Adult C/C-allele miceand Neonatal Δ7 Mouse Model
Preparation Method	Adult C/C-allele mice with mild SMA phenotype were treated for 10 days withRisdiplam (RG7916) given once a day orally at three different doses (1, 3, and 10 mg/kg). The level of SMN protein was assessed in brain and in quadriceps muscle. In the Δ7 mouse model of severe SMA, Risdiplam (RG7916) was administered by intraperitoneal (ip) injection once daily starting on postnatal day (PND) 3 and continued through PND9. The level of SMN protein was assessed in brain and in quadriceps muscle as well.
Dosage form	Adult C/C-allele mice：1, 3, and 10 mg/kg;10 days;p.o. Δ7 mouse model：0-3mg/kg;6 days;i.p.
Applications	Risdiplam (RG7916) potently increases SMN protein in both brain and muscle tissues of transgenic mouse models of SMA.
References: [1].Ratni H, Ebeling M,et,al. 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 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25. PMID: 30044619.

Risdiplam is a small molecule SMN2 pre-mRNA splicing modifier that promotes the inclusion of exon 7 and production of full-length SMN2 mRNA, which can compensate for the loss of SMN1 ^[1-2].

Risdiplam (RG7916)(0-1µM) was active in vitro in SMA patient-derived fibroblasts and in motor neurons generated from induced pluripotent stem cells (iPSCs) derived from SMA type 1 patient fibroblasts, promoting the inclusion of exon 7, to generate full-length (FL) mRNA^[1].

Risdiplam (RG7916) potently increases SMN protein in both brain and muscle tissues of transgenic mouse models of SMA^[1]. In infants with type 1 spinal muscular atrophy, treatment with oral risdiplam(0.08-0.2mg/kg/day)led to an increased expression of functional SMN protein in the blood[3].

References:
[1]. Ratni H, Ebeling M,et,al. 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 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25. PMID: 30044619.
[2]. Singh RN, Ottesen EW, et,al. The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy. Neurosci Insights. 2020 Nov 23;15:2633105520973985. doi: 10.1177/2633105520973985. PMID: 33283185; PMCID: PMC7691903.
[3]. Baranello G, Darras BT, et,al. FIREFISH Working Group. Risdiplam in Type 1 Spinal Muscular Atrophy. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. PMID: 33626251.

Cas No.	1825352-65-5	SDF
同義語	RG-7916, RO7034067
Canonical SMILES	O=C(C=C(C(C=C1C)=NN2C1=NC(C)=C2)N=C3C=C4)N3C=C4N(CCN5)CC65CC6
Formula	C₂₂H₂₃N₇O	M.Wt	401.46
溶解度	Ethanol : < 1 mg/mL (insoluble);DMSO : < 1 mg/mL (insoluble or slightly soluble)	Storage	Store at -20°C
General tips	Please select the appropriate solvent to prepare the stock solution according to the solubility of the product in different solvents; once the solution is prepared, please store it in separate packages to avoid product failure caused by repeated freezing and thawing.Storage method and period of the stock solution: When stored at -80°C, please use it within 6 months; when stored at -20°C, please use it within 1 month. To increase solubility, heat the tube to 37°C and then oscillate in an ultrasonic bath for some time.
Shipping Condition	Evaluation sample solution: shipped with blue ice. All other sizes available: with RT, or with Blue Ice upon request.

Complete Stock Solution Preparation Table

Prepare stock solution
		1 mg	5 mg	10 mg
	1 mM	2.4909 mL	12.4545 mL	24.9091 mL
	5 mM	0.4982 mL	2.4909 mL	4.9818 mL
	10 mM	0.2491 mL	1.2455 mL	2.4909 mL

モルアリティ計算機
希釈計算機

計算

In vivo Formulation Calculator (Clear solution)

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

Dosage: mg/kg

Average weight of animals: g

Dosing volume per animal:μL

Number of animals:

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+ %

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.
3. All of the above co-solvents are available for purchase on the GlpBio website.

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Review for Risdiplam (RG7916)

Average Rating: 5 ★★★★★ (Based on Reviews and 21 reference(s) in Google Scholar.)

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Risdiplam (RG7916) (Synonyms: RG-7916, RO7034067)

顧客レビュー

GlpBio Citations

Bioactive Compounds Premium Provider

評判の良い論文で引用されたGlpBio製品

Product Documents

Quality Control & SDS

Protocol

Complete Stock Solution Preparation Table

モルアリティ計算機

希釈計算機

In vivo Formulation Calculator (Clear solution)

レビュー

Risdiplam (RG7916) (Synonyms: RG-7916, RO7034067)

顧客レビュー

GlpBio Citations

Bioactive Compounds Premium Provider

評判の良い論文で引用されたGlpBio製品

Product Documents

Quality Control & SDS

Protocol

Complete Stock Solution Preparation Table

モルアリティ計算機

希釈計算機

In vivo Formulation Calculator (Clear solution)

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レビュー