Branaplam(LMI070)是一种高效，选择性和口服活性的小分子SMN2剪接调节剂。
CAS：1562338-42-4
分子式：C22H27N5O2
分子量：393.48
纯度：98%
存储：Store at -20°C

Background:

Branaplam (LMI070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

[1]. Palacino J, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-517.