Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM). branaplam hydrochloride inhibits hERG (IC50: 6.3 uM). In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride increased full-length SMN protein and improved survival.