The myotonic dystrophy type 1 drug development pipeline: 2026 Edition

• Myotonic dystrophy type 1 clinical development has accelerated, with multiple late-stage candidates. • Nucleic acid therapies emerge as leading candidates for myotonic dystrophy type 1 modification. • Therapeutic strategies diversify across multiple modalities. • Exercise and rehabilitation provide emerging nonpharmacological benefits in myotonic dystrophy type 1. • Myotonic dystrophy type 1 pipeline enters an unprecedented phase of maturity. Over the past few years, therapeutic development for myotonic dystrophy type 1 has accelerated markedly, with ongoing clinical trials nearly doubling and several candidates advancing to late-stage evaluation. Approaches have diversified, including small molecules, antibody–oligonucleotide conjugates, enhanced-delivery antisense platforms, gene therapy and miRNA-targeting strategies. Several nucleic-acid-based therapies demonstrate robust target engagement and clinically meaningful signals, positioning this modality as highly promising for disease modification. Parallel preclinical research has expanded the range of mechanisms under investigation, while structured exercise and rehabilitation continue to emerge as complementary nonpharmacological interventions. Together, these developments illustrate a therapeutic landscape entering an unprecedented phase of maturity, with multiple avenues progressing simultaneously toward potential clinical translation.