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The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review.

Shelagh M SzaboRenna M SalhanyAlison DeightonMeagan HarwoodJean MahKatherine L Gooch
Published in: Orphanet journal of rare diseases (2021)
Contemporary natural history studies from North America report that LOA on average occurs in the early teens, need for ventilation and cardiomyopathy in the late teens, and death in the third or fourth decade of life. Variability in rates may be due to differences in study design, treatment with corticosteroids or other disease-modifying agents, variations in clinical practices, and dystrophin mutations. Despite challenges in synthesizing estimates, these findings help characterize disease progression among contemporary North American DMD patients.
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