In the last few years, some of these compounds have shown significant activity against DM1 pathogenetic repeats through various mechanisms such as the inhibition of DNA transcription by binding CTG repeats, degradation of the toxic mutant transcript, release of MBNL from the ribonuclear foci through inhibition of MBNL:CUG transcript interaction, and lastly modulation of signaling pathways of downstream repeated RNA expression [85]. The gene discussed is MBNL1; the disease is myotonic dystrophy type 1.