Duchenne muscular dystrophy is a genetic disorder linked to the X-chromosome. It is the most common form of muscular dystrophy in children and occurs when the protein dystrophin is missing. This causes a gradual breakdown of muscles and a decline in muscle strength. Duchenne muscular dystrophy mainly affects boys. An estimated one in 3,500 males worldwide has the disorder, and each year approximately 400 boys in the United States are born with it. Symptoms usually appear between ages two and five and include frequent falls, large calf muscles, and difficulty running, jumping, and getting up. There is no cure.
The guideline authors reviewed all available research for the use of corticosteroids in the treatment of Duchenne muscular dystrophy. Corticosteroids are man-made drugs that are similar to the body's hormone cortisone. Two corticosteroids, prednisone and deflazacort, were found to slow the rate of muscle deterioration, and are recommended as potential treatments to minimize the effect of Duchenne muscular dystrophy.
Prednisone was found to help muscle strength and function and should be offered as a treatment option. Deflazacort, a drug similar to prednisone, is also recommended as a treatment option but is not available in the United States at this time.
"Corticosteroids are the only effective drugs in providing improvements in children with Duchenne muscular dystrophy," said lead author Richard T. Moxley III, MD, of the University of Rochester in Rochester, N.Y.