Biological Activity
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.
In Vitro
No in vitro data are provided in the current product source. Contact us for further information.
In Vivo
No in vivo data are provided in the current product source. Contact us for further information.
References & source
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
Published experimental findings depend on the model and assay conditions. They do not guarantee equivalent performance in every application. For laboratory research use only.