For research use only. Not for therapeutic Use.
Branaplam hydrochloride（Cat No.:I023653）is a small molecule inhibitor of the splicing factor RNA binding protein, a key regulator in the production of survival motor neuron (SMN) protein. It has shown potential as a therapeutic agent for spinal muscular atrophy (SMA), a genetic disorder characterized by the loss of motor neurons and muscle weakness. By modulating the splicing of the SMN2 gene, Branaplam hydrochloride increases the production of functional SMN protein, thereby improving motor function and slowing disease progression. It is currently under investigation in clinical trials for its efficacy and safety in SMA treatment.
| CAS Number | 1562338-39-9 |
| Synonyms | 5-(1H-pyrazol-4-yl)-2-[6-(2,2,6,6-tetramethylpiperidin-4-yl)oxypyridazin-3-yl]phenol;hydrochloride |
| Molecular Formula | C22H28ClN5O2 |
| Purity | ≥95% |
| IUPAC Name | 5-(1H-pyrazol-4-yl)-2-[6-(2,2,6,6-tetramethylpiperidin-4-yl)oxypyridazin-3-yl]phenol;hydrochloride |
| InChI | InChI=1S/C22H27N5O2.ClH/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);1H |
| InChIKey | XJIMIVJABPKGIY-UHFFFAOYSA-N |
| SMILES | CC1(CC(CC(N1)(C)C)OC2=NN=C(C=C2)C3=C(C=C(C=C3)C4=CNN=C4)O)C.Cl |
| Chemistry Calculators | Dilution Calculator In vivo Formulation Calculator Molarity Calculator Molecular Weight Calculator Reconstitution Calculator |
