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.
MedKoo Cat#: 526830
Name: ML372
CAS#: 1331745-61-9
Chemical Formula: C18H20N2O4S
Exact Mass: 360.1144
Molecular Weight: 360.43
Elemental Analysis: C, 59.98; H, 5.59; N, 7.77; O, 17.76; S, 8.89
The following data is based on the product molecular weight 360.43 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
---|---|---|---|
1 mM | 1.15 mL | 5.76 mL | 11.51 mL |
5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
50 mM | 0.02 mL | 0.12 mL | 0.23 mL |