Branaplam hydrochloride

Biorbyt

Branaplam hydrochloride

10

Research Use Only

C22H28CLN5O2

429.94

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 microM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.

OC1C=C(C2=CNN=C2)C=CC=1C1N=NC(OC2CC(C)(C)NC(C)(C)C2)=CC=1.Cl

12352200