Branaplam hydrochloride - CAS 1562338-39-9

Branaplam hydrochloride (LMI070; NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 μM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival.

Others, DNA/RNA Synthesis, Potassium Channel

DNA Damage/DNA Repair, Membrane transporter/Ion channel, Cell Cycle/Checkpoint, Others

1562290

0.9845

C22H28ClN5O2

429.95

Cl.OC1=CC(=CC=C1C=2N=NC(OC3CC(NC(C)(C)C3)(C)C)=CC2)C=4C=NNC4

-20°C

Ice Packs

Small Compound