Importantly, the observed beneficial effects of ACT and erythromycin in DM1 results from interference with the underlying molecular pathology of the disease where ACT exerts its therapeutic effects by reducing CUG-RNA transcript level [14] whereas erythromycin by direct inhibition of MBNL1 sequestration via competitive binding to the expanded CUG repeats [16]. Here, MBNL1 is linked to myotonic dystrophy type 1.