Duchenne Muscular Dystrophy (DMD) is a disease that makes muscles weak over time. This study is checking how safe and helpful a new medicine, DYNE-251, is for boys aged 4-16 who have a specific gene problem that can be treated with exon 51 skipping. Exon 51 skipping is a way to make muscle proteins work better. The study lasts 240 weeks (about 4.6 years) and has three parts: the first is 24 weeks with some receiving a placebo (a fake treatment), the second is openly giving DYNE-251 for 24 weeks, and the last is a long-term follow-up for 192 weeks. Participants must be able to have a muscle biopsy and need to have stable heart and lung function. They cannot have had certain treatments, like gene therapy, recently.
- Duration: Up to 240 weeks with regular visits.
- Participants receive multiple doses of DYNE-251.
- Must be able to handle muscle biopsy and stable medication dose.
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