Branaplam hydrochloride

• Catalog Number: orb1473134
• Category: Small Molecules
• Description: Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.
• CAS Number: [1562338-39-9]
• MW: 429.94
• SMILES: OC1C=C(C2=CNN=C2)C=CC=1C1N=NC(OC2CC(C)(C)NC(C)(C)C2)=CC=1.Cl
• Formula: C₂₂H₂₈CLN₅O₂
• Note: For research use only
• Expiration Date: 12 months from date of receipt.