The pathophysiology of DM1 is extremely complex, and a bundle of small molecule compounds such as up-regulators of the muscleblind-like (MBNL) splicing factor family, H-RAS pathway inhibitors, transcription inhibitors, and protein kinase modulators has been shown to mitigate DM1 pathogenesis in different experimental systems (48), giving hope that more clinical trials will start in the nearer future (45). The gene discussed is SLU7; the disease is myotonic dystrophy type 1.