ML372
ML372 is a potent and selective small molecule SMN2 splicing modulator, exhibiting an EC50 of 12 nM and increasing SMN protein levels by 325%. It demonstrates favorable pharmacokinetics, blood-brain barrier penetration, and tolerability, supporting its use in preclinical research for spinal muscular atrophy (SMA) in both cellular and animal models.
Product Specifications
CAS Number
1331745-61-9
Field of Research
Cell Biology, Molecular Biology
Smiles
OC (=O) C1CCN (CC1) c1ncc (s1) -c1ccc2OCCCOc2c1
Molecular Formula
C18H20N2O4S
Molecular Weight
360.43
Storage Conditions
-20°C
Notes
For research use only.
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