New research has shown that the corticosteroid deflazacort is a safe and effective treatment for Duchenne muscular dystrophy. The findings, which were published online on August 26, 2016 in the journal Neurology, could pave the way for first U.S.-approved treatment for the disease. "Duchenne muscular dystrophy patients have limited treatment options and a desperate need for effective therapies," said University of Rochester Medical Center (URMC) neurologist Robert Griggs, M.D., lead author of the study. "This study shows that deflazacort may provide an important treatment for delaying the progression of the disease." Duchenne muscular dystrophy (DMD) is a condition found almost exclusively in boys. The disease is characterized by muscle weakness which begins to appear at a young age and progresses rapidly leading to significant disability. Boys with the disease often end up in a wheelchair by age 9 or 10 because of weakness in their legs. The symptoms eventually spread to other parts of the body, including the heart and muscles responsible for breathing, and the disease is often fatal by the time the individual reaches his late teens. An estimated 28,000 people in the U.S. suffer from the disease. While there is currently no approved treatment for DMD, the corticosteroid prednisone is often used "off label" to treat the condition. Several studies, beginning with research conducted by Dr. Griggs and his colleagues more than 20 years ago, have shown that daily use of corticosteroids can increase muscle mass and slow muscle degeneration in DMD patients, prolonging their ability to walk and preserving respiratory function. However, many DMD patients in the U.S. are not prescribed corticosteroids, primarily due to concern over the side effects of prolonged drug use in children.
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