ML372

ML372 is a small molecule SMN modulator that increases SMN protein in patient fibroblasts with EC50 of 37 nM; possesses good potency, pharmacokinetics, tolerance, and CNS penetration that are able to increase levels of SMN protein in several model cell lines; increase SMN protein levels in vivo, restore motor function, and prolong survival of SMNΔ7 SMA Mice; improves the righting reflex and extended survival of a severe mouse model of SMA.

References 1. Burnett BG, et al. Probe Reports from the NIH Molecular Libraries Program [Internet]. 2. Abera MB, et al. JCI Insight. 2016 Nov 17;1(19):e88427. View Related Products by Target SMN2 Modulator