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Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

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

Published in the Journal of Comparative Effectiveness Research, this paper compares the effectiveness of corticosteroids by age and disease progression across patients from a range of Phase 2b and Phase 3 clinical trials

  • Review patient demographics from 4 clinical trials
  • Compare the efficacy results for 2 different corticosteroid treatments on disease progression through measures such as 6-minute walk distance and North Star Ambulatory Assessment scores
  • Understand the impact of corticosteroid treatment on motor function from baseline to 48 weeks
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-DFZ-2500002 | March 2025

McDonald CM, Marden JR, Shieh PB, et al. J Comp Eff Res. 2023;12(4):e220190

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MED-ALL-DMD-2300173 | June 2025

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