Abstract
Deflazacort (Emflaza™) is a derivative of prednisolone with immunomodulatory and anti-inflammatory properties. Overall, in the treatment of boys with Duchenne muscular dystrophy (DMD), deflazacort is as effective as prednisone, but may be better tolerated. In a randomized, double-blind, phase 3 trial in boys with DMD, deflazacort and prednisone both preserved muscle strength more effectively than placebo after 12 weeks of treatment, with improvements being maintained until study end at week 52. Both active treatments were also associated with improved motor function. In the real-word setting, treatment with corticosteroids for ≥ 1 year significantly delayed disease progression relative to no or < 1 month of corticosteroid treatment, with deflazacort showing some benefits over prednisone/prednisolone. Body weight gain-related events were less frequent with deflazacort than with prednisone, but stunted growth and the development of non-serious cataracts were more frequent.
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