Confirmatory studies revealed promising drug candidates for DM1 that acted through separate mechanisms of action, such as the reduction of aberrant ribonuclear aggregates (foci in which MBNL1 is sequestered by the expanded CUG-RNAs) or the ability to bind toxic CUG-RNA molecules, which indirectly modified the splicing read-out. Here, MBNL1 is linked to myotonic dystrophy type 1.