Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Functional and clinical outcomes associated with steroid treatment among non-ambulatory patients with Duchenne muscular dystrophy

Publication: Functional and clinical outcomes associated with steroid treatment among non-ambulatory patients with Duchenne muscular dystrophy

Published in the Journal of Neuromuscular Diseases, this paper compares the effectiveness of 2 different corticosteroids in preserving limb function in non-ambulatory patients with Duchenne muscular dystrophy (DMD)

Learn about the efficacy of deflazacort and prednisone in non-ambulatory adolescent patients with DMD
Review how different corticosteroid treatments can affect disease progression milestones, including upper limb performance
Understand the impact of corticosteroid treatment on cardiac and pulmonary function through measures such as forced vital capacity and cough peak flow

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

McDonald CM, Mayer OH, Hor KN, et al. J Neuromuscul Dis. 2023;10(1):67–79

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MED-ALL-DMD-2300173 | June 2025

Publication: Functional and clinical outcomes associated with steroid treatment among non-ambulatory patients with Duchenne muscular dystrophy

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