Weekend high-dosage prednisone
A new option for treatment of Duchenne muscular dystrophy
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In 2005, the American Academy of Neurology published a practice parameter entitled “Corticosteroid treatment of Duchenne dystrophy.”1 That report concluded that prednisone (0.75/mg/kg/day) and deflazacort (0.9 mg/kg/day) represented optimal dosages of corticosteroid treatment, that daily was better than alternate day therapy with prednisone, and that both corticosteroids produced similar beneficial effects on muscle strength and function and caused similar side effects that patients tolerated without major problems.1 In the closing section of that practice parameter, there were 10 recommendations for future research. The second, sixth, and seventh of these recommendations encouraged investigators to determine if daily corticosteroid treatment with prednisone and deflazacort exerts a beneficial effect on cardiac and respiratory function, whether long-term benefits occur to ambulation, respiratory, and cardiac function, and whether the long-term side effects are acceptable.1 Four subsequent publications addressed these 3 recommendations.2,–,5 They report that daily prednisone and deflazacort given for over 5 years prolongs ambulation by 2–5 years, reduces the need for spinal stabilization surgery, improves cardiopulmonary function, …
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