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.
Molecular Weight:
432.35
Purity:
99.58%
CAS Number:
[1005504-62-0]
Formula:
C21H23Cl2N5O
Target:
DNA/RNA Synthesis|||Others
T3336
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