Publication: Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial
This research article, published in the Muscle & Nerve Journal, details a post hoc analysis of the placebo arm of the Ataluren Confirmatory Trial (ACT) Duchenne muscular dystrophy (DMD) study
Review the efficacy and safety of deflazacort compared with prednisone/prednisolone evaluated in a post hoc analysis of the placebo arm of the ACT DMD trial in patients with nonsense mutation DMD
Endpoints including change from baseline in 6‐minute walk distance (6MWD), timed function tests, estimated age at loss of ambulation (extrapolated from 6MWD) are discussed
Shieh PB, Mcintosh J, Jin F, et al. Muscle Nerve. 2018;58(5):639–645
Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. PTC Therapeutics is the Marketing Authorization Holder for deflazacort in the US only. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The US Prescribing Information for deflazacort is available here. Please refer to your local country guidance for more information.
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MED-ALL-DMD-2200190 | August 2023
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