Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

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...

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Bibliographic Details

Published in:	Journal of Comparative Effectiveness Research
Main Authors:	Craig M McDonald, Jessica R Marden, Perry B Shieh, Brenda L Wong, Henry Lane, Adina Zhang, Ha Nguyen, Molly Frean, Panayiota Trifillis, Karyn Koladicz, James Signorovitch
Format:	Article
Language:	English
Published:	Becaris Publishing Limited 2023-02-01
Subjects:	clinical outcomes deflazacort disease milestones duchenne muscular dystrophy efficacy prednisone/prednisolon