Long-term benefits of glucocorticoids in Duchenne muscular dystrophy: is it worth it?


The CINRG Duchenne muscular dystrophy (DMD) Natural History Study (DNHS) examined the long-term benefits of glucocorticoids (GC) based on milestone-related disease progression across the lifespan and risk of death. The 10-year prospective DNHS followed 440 males ages 2–28 years with DMD comparing none or < 1 month and 3 1 year cumulative GC treatment with regard to progression to 8 disease-related and clinically meaningful mobility and upper limb milestones. Kaplan-Meier survival analyses compared GC treatment groups for time to stand from supine 3 5 and 3 10 seconds, and loss of: stand from supine, 4-stair climb, ambulation, full overhead reach, hand to mouth, and hand function.


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