Finding the Optimum Regimen for Duchenne Muscular Dystrophy

Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up. Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems. Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all. The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects: 1. Prednisone 0.75mg/kg/day 2. Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment 3. Deflazacort 0.9mg/kg/day. The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.