Branaplam [CAS 1562338-42-4]

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].

[1562338-42-4]

C22H27N5O2

393.48

OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3

DNA/RNA Synthesis; Potassium Channel

99.78

DMSO : 3.33 mg/mL (ultrasonic; warming; heat to 60°C)

4°C (Powder, sealed storage, away from moisture)

Room Temperature

H302, H312, H332

H302, H312, H332