LDN193189 Tetrahydrochloride is a selective BMP type I receptor inhibitor, which efficiently inhibits ALK2 and ALK3 (IC50=5 nM and 30 nM, respectively), with weaker effects on ALK4, ALK5 and ALK7 (IC50≥500 nM)[1].
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.