Adrenal Corticosteroids Provide Long-Term Benefit in Duchenne Muscular Dystrophy

In the largest study to date of adrenal corticosteroid treatment of Duchenne muscular dystrophy, investigators detail the relative preservation of upper- and lower-extremity strength and respiration and prolongation of life.

In this multicenter study, researchers observed outcomes in 339 patients with Duchenne muscular dystrophy (DMD) treated with variable regimens of steroids for ≥1 year and 69 patients who were not steroid-treated. The investigators assessed the effects of steroid treatment on the age when each of 8 functional “milestones” was lost (e.g., time to stand from supine position), reduction of pulmonary function, and death in subgroups of steroid-treated versus untreated DMD patients.

Death and all but one of the milestones (ability to climb 4 steps) were significantly delayed in steroid-treated patients. Deflazacort was superior to prednisone for several milestones.

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The current finding of beneficial effects of glucoco…

Reviewing Author

Daniel B. Drachman, MD

Adrenal Corticosteroids Provide Long-Term Benefit in Duchenne Muscular Dystrophy

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Reviewing Author