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Description: Rg3039 (PF-06687859) is a potent DcpS inhibitor. DcpS is a therapeutic target for spinal muscular atrophy. Spinal muscular atrophy (SMA) is caused by deletion or mutation of both copies of the SMN1 gene which produces an essential protein known as SMN. RG3039 improves motor function in SMA mice. RG3039 also showed activity to improve survival, function and motor unit pathologies in two SMA mouse models. Target: Others. Smiles: Nc1nc(N)c2c(OCC3CCN(Cc4c(Cl)cccc4Cl)CC3)cccc2n1. References: Rocky G. Gogliotti, et al. Hum Mol Genet. 2013 Oct 15, 22(20): 4084-4101.
This website uses cookies, which are necessary for the technical operation of the website and are always set. Other cookies, which increase the usability of this website, serve for direct advertising or simplify interaction with other websites and social networks, will only be used with your consent.
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