In summary, our results revealed that the MP compounds, a new family of FKBP12 ligands, rescued multiple and critical DM1 phenotypes in two different models of the disease: elevated ROS production, impaired proliferation and metabolism in human primary fibroblasts in vitro; and reduced longevity and impaired locomotor activity in a Drosophila model in vivo. The gene discussed is FKBP1A; the disease is myotonic dystrophy type 1.