1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis

Risdiplam (Synonyms: RG7916; RO7034067)

Cat. No.: HY-109101 Purity: 99.31%
Handling Instructions

Risdiplam (RG7916; RO7034067) 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.

Risdiplam Chemical Structure

Risdiplam Chemical Structure

CAS No. : 1825352-65-5

Size Price Stock Quantity
5 mg USD 650 In-stock
Estimated Time of Arrival: December 31
10 mg USD 990 In-stock
Estimated Time of Arrival: December 31
25 mg USD 1990 In-stock
Estimated Time of Arrival: December 31
50 mg   Get quote  
100 mg   Get quote  

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Description

Risdiplam (RG7916; RO7034067) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels.

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].

Clinical Trial
Solvent & Solubility
In Vitro: 

Ethanol : < 1 mg/mL (insoluble)

DMSO : < 1 mg/mL (insoluble or slightly soluble)

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 0.4982 mL 2.4909 mL 4.9818 mL
10 mM 0.2491 mL 1.2455 mL 2.4909 mL
*Please refer to the solubility information to select the appropriate solvent.
References
Molecular Weight

401.46

Formula

C₂₂H₂₃N₇O

CAS No.

1825352-65-5

SMILES

O=C(C=C(C(C=C1C)=NN2C1=NC(C)=C2)N=C3C=C4)N3C=C4N(CCN5)CC65CC6

Storage
Powder -20°C 3 years
  4°C 2 years
In solvent -80°C 6 months
  -20°C 1 month
Shipping

Room temperature in continental US; may vary elsewhere

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Product Name:
Risdiplam
Cat. No.:
HY-109101
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Risdiplam

Cat. No.: HY-109101