SMN2 选择性调节剂

目录号 产品名 产品描述 Selective / Pan IC50 / Ki
S6642 Branaplam (LMI070)

Branaplam (LMI070) is a highly selective, small-molecule splicing modulators of survival motor neuron‑2 (SMN2) with an EC50 of 0.02 μM.

Selective SMN2, EC50: 0.02 μM
S6925 Risdiplam (RG7916)

Risdiplam (Evrysdi, RG7916, RO7034067) is an orally administered SMN2 pre-mRNA splicing modifier that distributes into the central nervous system (CNS) and peripheral tissues.