Branaplam;LMI070; NVS-SM1 hydrochloride

CAS NO.:1562338-39-9

Molecular Formula:C₂₂H₂₈ClN₅O₂

Molecular Weight:429.94

Purity: > 98%

Solubility (R.T.: 25℃): DMSO

Stability: - 20℃ 2 years

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. It 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.

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