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Abstract #3833

Deflazacort & prednisone/prednisolone treatment in Duchenne muscular dystrophy: Disease trajectory differences modeled using MR biomarkers

Ishu Arpan1, William D Rooney1, Rebecca J Willcocks2, Alison M Barnard2, Sean C Forbes2, William T Triplett2, Eric Baetscher1, Michael J Daniels2, Glenn A Walter2, and Krista Vandenborne2
1Oregon Health & Science University, Portland, OR, United States, 2University of Florida, Gainesville, FL, United States

Duchenne muscular dystrophy (DMD) is a genetic disease characterized by rapidly progressing weakness and degeneration of the skeletal muscles. This study presents a modeling approach using MR biomarkers to characterize disease progression in individuals with DMD receiving deflazacort, prednisone/prednisolone, or no corticosteroid treatment. The findings from a sample of convenience compiled from the ImagingDMD study demonstrate that 1) corticosteroid treatment slows DMD disease progression, with deflazacort slowing progression to a greater extent than prednisone/prednisolone, and 2) the slow-progressing muscles in DMD are more sensitive to treatment effects, and hence, could be better candidates to assess therapeutic response in clinical trials.

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