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- W4319332816 abstract "Aim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. Methods: Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment total score. Results: Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (>3 years). Conclusion: Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients." @default.
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- W4319332816 date "2023-02-07" @default.
- W4319332816 modified "2023-09-30" @default.
- W4319332816 title "Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status" @default.
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- W4319332816 doi "https://doi.org/10.57264/cer-2022-0190" @default.
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