Rg3039 - CAS 1005504-62-0

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.

Others, DNA/RNA Synthesis

Cell Cycle/Checkpoint, Others, DNA Damage/DNA Repair

1005442

0.9958

C21H23Cl2N5O

432.35

Nc1nc(N)c2c(OCC3CCN(Cc4c(Cl)cccc4Cl)CC3)cccc2n1

-20°C

Ice Packs

Small Compound