Description
RG3039 is a potent DcpS inhibitor. It is used for the treatment for spinal muscular atrophy(SMA), which is caused by deletion or mutation of both copies of the SMN1 gene. It improves motor function in SMA mice. It also showed activity to improve survival, function and motor unit pathologies in two SMA mouse models. It was developed by Repligen and now in Clinic Phase 1 with no progress