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Risdiplam

Catalog No. T16757 CAS 1825352-65-5

Synonyms: RO7034067, RG7916

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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Risdiplam, CAS 1825352-65-5

Pack Size	Availability	Price/USD
1 mg	In stock	$ 55.00
2 mg	In stock	$ 81.00
5 mg	In stock	$ 129.00
10 mg	In stock	$ 211.00
25 mg	In stock	$ 376.00
50 mg	In stock	$ 559.00
100 mg	In stock	$ 786.00

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Purity: 99.43%

Purity: 99.36%

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Description

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].

Synonyms	RO7034067, RG7916
Molecular Weight	401.46
Formula	C22H23N7O
CAS No.	1825352-65-5

Storage

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

Solubility Information

DMSO: 2 mg/mL (4.98 mM), Sonication is recommended.

Ethanol: 2 mg/mL (4.98 mM), Sonication is recommended.

H2O: 2.22 mg/mL (5.53 mM), when pH is adjusted to 6 with HCl. Sonication is recommended.

References and Literature

1. Poirier A, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447.

Related compound libraries

This product is contained In the following compound libraries：

EMA Approved Drug Library Anti-Cancer Approved Drug Library Anti-Cancer Drug Library Drug Repurposing Compound Library FDA-Approved & Pharmacopeia Drug Library Bioactive Compound Library Bioactive Compounds Library Max Human Metabolite Library Cell Cycle Compound Library FDA-Approved Drug Library

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Keywords

Risdiplam 1825352-65-5 Cell Cycle/Checkpoint DNA Damage/DNA Repair DNA/RNA Synthesis RG 7916 Inhibitor RO 7034067 RO7034067 RG-7916 RO-7034067 inhibit RG7916 inhibitor

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