Branaplam (LMI070, NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 µM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model[1][2].
Molecular Weight:
393.48
Purity:
99.78
CAS Number:
[1562338-42-4]
Formula:
C22H27N5O2
Target:
DNA/RNA Synthesis,Potassium Channel
Application Notes:
MCE Product type: Reference compound
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