NVS-SM1 --- SMN2 Splicing Modulator
Overview | |
Catalog # | bs-60277c-2-mg-solid |
Product Name | NVS-SM1 --- SMN2 Splicing Modulator |
Specifications | |
Storage Buffer | Powder |
Storage Condition | Store in dry, dark place at -20C for 1 year. |
Target | |
Product Information | Molecular Weight: 393.48 Formula: C22 H27 N5 O2 CAS Number: 1562338-42-4 InChi Key: STWTUEAWRAIWJG-UHFFFAOYSA-N InChi: InChI=1S/C22H27N5O2/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15/h5-9,12-13,16,27-28H,10-11H2,1-4H3,(H,23,24) Smiles: CC1(C)CC(CC(C)(C)N1)OC1=CC=C(N=N1)C1=CC=C(C=C1O)C1C=NNC=1 Purity: 98.0 Solubility: DMSO: 3 mg/mL(7.62 mM). Water: Insoluble. Appearance: Solid Power. Shelf Life: 1.0 years |
Description | NVS-SM1, also known as Branaplam and LMI-070, is a highly potent, selective and orally active small molecule SMN2 splicing modulator as a potential treatment for SMA Type 1 (Spinal Muscular Atrophy) .NVS-SM1 works by increasing the amount of functional SMN protein produced by the “back-up” gene, SMN2, through modifying its splicing. |