ML372 is a potent and selective SMN Modulator (EC50 = 12 nM, 325% increase inSMN2). ML372 Increases SMN Protein Abundance, Body Weight, Lifespan, and Rescues Motor Function in SMNΔ7 SMA Mice. possessed a more stable half-life in mouse liver microsomes (T1/2 > 60 min) and high permeability in Caco-2 cells (Papp A to B = 33.8 × 10–6 cms–1 and Papp B to A = 44.1 × 10–6 cms–1) with an efflux ratio close to unity (i.e., 1.3). ML372 shows good potency, pharmacokinetics, tolerance, and CNS penetration that are able to increase levels of SMN protein in several model cell lines. Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease and the most common inherited cause of infant mortality. SMA, with a carrier frequency of ~1:40, affects ~ 1:8,000 births. References: Burnett BG, Xiao J, Southall N, Zheng W, Ferrer M, Cherry JJ, Androphy EJ, Fischbeck K, Marugan JJ. SMN Modulator ML372 Increases SMN Protein Abundance, Body Weight, Lifespan, and Rescues Motor Function in SMNΔ7 SMA Mice. 2013 Dec 15 [updated 2015 Feb 11]. Probe Reports from the NIH Molecular Libraries Program [Internet]. Bethesda (MD): National Center for Biotechnology Information (US); 2010-. Available from .gov/books/NBK280051/ PubMed PMID: 25834905.

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CAS：1331745-61-9