Description
Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels.
Product information
CAS Number: 1825352-65-5
Molecular Weight: 401.46
Formula: C22H23N7O
Chemical Name: 7-{4,7-diazaspiro[2.5]octan-7-yl}-2-{2,8-dimethylimidazo[1,2-b]pyridazin-6-yl}-4H-pyrido[1,2-a]pyrimidin-4-one
Smiles: CC1=CC(=NN2C=C(C)N=C21)C1=CC(=O)N2C=C(C=CC2=N1)N1CC2(CC2)NCC1
InChiKey: ASKZRYGFUPSJPN-UHFFFAOYSA-N
InChi: InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3
Technical Data
Appearance: Solid Power
Purity: ≥98% (or refer to the Certificate of Analysis)
Solubility: DMSO : 2 mg/mL (4.98 mM; Need ultrasonic). H2O : 1 mg/mL (2.49 mM; ultrasonic and warming and heat to 80°C).
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis
Storage Condition: Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.
Shelf Life: ≥12 months if stored properly.
Stock Solution Storage: 0 - 4 oC for 1 month or refer to the Certificate of Analysis.
Drug Formulation: To be determined
HS Tariff Code: 382200
How to use
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.
Products are for research use only. Not for human use.
Payment & Security
Your payment information is processed securely. We do not store credit card details nor have access to your credit card information.