Myotonic myopathy refers to a group of inherited muscle disorders associated with clinical and/or electrical myotonia. Conventional treatments primarily include pharmacotherapy (e.g., mexiletine) and non-pharmacological interventions (e.g., aerobic exercise, strength training), which are largely symptomatic and lack disease-modifying therapies. In recent years, emerging technologies in the biomedical field have continued to evolve, with targeted therapies represented by antisense oligonucleotide drugs and gene editing becoming research hotspots. This article summarizes the therapeutic research on myotonic myopathy conducted both domestically and internationally, and discusses future research directions in this field.
