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.

Providing storage is as stated on the product vial and the vial is kept tightly sealed, the product can be stored for up to 24 months.
Wherever possible, you should prepare and use solutions on the same day. However, if you need to make up stock solutions in advance, we recommend that you store the solution as aliquots in tightly sealed vials at -20°C. Generally, these will be useable for up to one month. Before use, and prior to opening the vial we recommend that you allow your product to equilibrate to room temperature for at least 1 hour.

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.