Duchenne muscular dystrophy (DMD) is a serious condition without a cure. Instead, treatments aim to slow down its progression. Currently, there are experimental treatments in phase I trials that could offer new options for patients.

This review discusses ongoing and recent early-stage trials for DMD treatments. These include therapies that skip certain genetic sections (exons) and gene therapies. Additionally, it covers phase I trials from the past five years involving various treatments like inhibitors, small molecules, synthetic flavanols, cell-based therapies, and repurposed medications.

Most recent trials have focused on antisense oligonucleotide (AON) treatments, which are already conditionally approved for DMD. Many of these trials involve exon-skipping AONs.

Recently, there's been interest in gene transfer therapies, like Elevidys, which delivers micro-dystrophin. However, their safety and effectiveness are still being studied in phase I trials. Other approaches in phase I trials vary in their effectiveness, safety, and how they're measured.