Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial

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

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.

For medical information, product complaints, or to report an adverse event, please call 1-866-562-4620 or email at usmedinfo@ptcbio.com. You may also report adverse events directly to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

MED-ALL-DFZ-2500002 | March 2025

Shieh PB, Mcintosh J, Jin F, et al. Muscle Nerve. 2018;58(5):639–645

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MED-ALL-DMD-2200190 | June 2025

Publication: Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial

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