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Risdiplam

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Purity:99.43%

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COA HPLC HNMR

Risdiplam

Catalog No. T16757Cas No. 1825352-65-5

Risdiplam (RO7034067) (RG7916) is orally administered. Risdiplam is a centrally and peripherally distributed SMN2 pre-mRNA splicing modifier which increases survival motor neuron (SMN) protein levels[1].

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Pack Size	Price	Availability
1 mg	$55	In Stock
2 mg	$81	In Stock
5 mg	$129	In Stock
10 mg	$211	In Stock
25 mg	$376	In Stock
50 mg	$559	In Stock
100 mg	$786	In Stock

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Product Introduction

Bioactivity

Description	Risdiplam (RO7034067) (RG7916) is orally administered. Risdiplam is a centrally and peripherally distributed SMN2 pre-mRNA splicing modifier which increases survival motor neuron (SMN) protein levels[1].
In vitro	Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full-length transcript and thus functional SMN protein. Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels [1]. 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]. Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full-length transcript and thus functional SMN protein. Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels [1]. 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].
Alias	RO7034067, RG7916

Chemical Properties

Molecular Weight	401.46
Formula	C22H23N7O
Cas No.	1825352-65-5

Storage & Solubility Information

Storage

Powder: -20°C for 3 years | In solvent: -80°C for 1 year

Solubility Information

DMSO: < 1mg/ml (insoluble）

Ethanol: 2 mg/mL (4.98 mM)

H2O: 2.22 mg/mL (5.53 mM)

Solution Preparation Table

H2O/Ethanol

	1mg	5mg	10mg	50mg
1 mM	2.4909 mL	12.4545 mL	24.9091 mL	124.5454 mL

H2O

	1mg	5mg	10mg	50mg
5 mM	0.4982 mL	2.4909 mL	4.9818 mL	24.9091 mL
10 mM	0.2491 mL	1.2455 mL	2.4909 mL	12.4545 mL
20 mM	0.1245 mL	0.6227 mL	1.2455 mL	6.2273 mL
50 mM	0.0498 mL	0.2491 mL	0.4982 mL	2.4909 mL
100 mM	0.0249 mL	0.1245 mL	0.2491 mL	1.2455 mL

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Mother liquor preparation method: 2 mg of drug dissolved in 50 μL DMSO TargetMol | reagent

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Preparation method for in vivo formula: Take 50 μL DMSO TargetMol | reagent

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mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLddH2O TargetMol | reagent

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