Emerging Investigational Therapies in Duchenne Muscular Dystrophy

Beyond currently approved therapies, several investigational approaches in Duchenne muscular dystrophy (DMD) are generating excitement for their potential to further alter disease progression. These include novel gene-editing strategies, such as CRISPR-based therapies, designed to correct dystrophin mutations at the genomic level, and next-generation exon-skipping agents aimed at expanding treatment eligibility to a broader patient population. Other promising avenues target secondary disease pathways, including fibrosis, inflammation, and muscle regeneration, with small molecules, biologics, and stem cell–based therapies under evaluation. Early clinical and preclinical data suggest these approaches may enhance muscle function, reduce tissue damage, and improve long-term outcomes. While challenges remain—such as optimizing delivery, ensuring safety, and determining long-term efficacy—these investigational therapies represent a shift toward precision, disease-modifying care in DMD, offering hope for patients and families seeking options beyond conventional steroids, small molecules, and first-generation gene therapies.