Therapeutic Innovation, Clinical Trial Acceleration, and Lifestyle Interventions in Myotonic Dystrophy Type 1: A Systematic Review

Therapeutic development in myotonic dystrophy type 1 (DM1) has accelerated in recent years, with a near doubling of clinical trials and expansion into late-stage development. The therapeutic landscape has diversified to include small molecules, oligonucleotide-based therapies, antibody-oligonucleotide conjugates, gene therapies, and microRNA-targeting strategies. Nucleic acid- based approaches have shown promising target engagement and early clinical signals, supporting their potential for disease modification. In parallel, preclinical avances. and non-pharmacological interventions such as exercise and rehabilitation are broadening the therapeutic scope. Overall, DM1 research is entering a more mature phase with multiole conversing therapeutic strategies.