Natural History of Duchenne Muscular Dystrophy

This study tracks the natural progression of Duchenne Muscular Dystrophy (DMD) in young boys over time. DMD is a genetic muscle-wasting disease that primarily affects males, causing progressive weakness that typically begins in the legs and spreads throughout the body. By carefully measuring how boys with DMD change over time — through walking tests, muscle assessments, and heart and lung checks — researchers can better understand the disease and establish benchmarks for evaluating future treatments. This is not a treatment trial; it is a natural history study. Participants receive standard care while researchers collect detailed measurements. Boys must be between 4 and 9 years old, have a genetic confirmation of DMD, be able to walk and pass certain movement tests, and be on corticosteroid therapy (the current standard of care). The study carefully excludes boys who have already received gene therapies or experimental DMD drugs to ensure the data reflects the true natural course of the disease. You may be eligible if: - Your son is a male between 4 and 9 years old - He has a confirmed genetic diagnosis of DMD - He can walk and achieve a North Star Ambulatory Assessment score above the threshold - He is on or starting corticosteroid therapy - His heart function is normal (ejection fraction at or above 55%) - He does not require breathing assistance You may NOT be eligible if: - Your son has significant heart problems (cardiomyopathy with ejection fraction below 55%) - He needs ventilator support - He has previously received gene therapy for DMD - He is currently enrolled in another interventional clinical trial Talk to your doctor to see if this trial is right for you.