Summary

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• Emflaza (deflazacort) is indicated to treat Duchenne muscular dystrophy in patients ≥ 2 years of age.
• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• Deflazacort demonstrated a significant improvement in a 6-minute walk distance with a least-squares mean difference of 39.54 meters (95% CI: 13.799, 65.286; p = 0.0026) and a lower decline of 28.3 meters (95% CI: 5.7, 50.9) compared to prednisone/prednisolone in ambulatory boys with nonsense mutation Duchenne muscular dystrophy (DMD).
• Deflazacort-treated patients experienced lower declines in timed function tests, including 2.9 seconds on rise from supine (95% CI: 0.9, 4.9 seconds) and 2.3 seconds on the 4-stair climb (95% CI: 0.5, 4.1 seconds), compared to prednisone/prednisolone.
• Deflazacort led to a lower decline of 2.9 points on the North Star Ambulatory Assessment linearized score (95% CI: 0.1, 5.8) in ambulatory boys with nonsense mutation DMD.
• No safety information was found in the reviewed studies.
• The studies focused on ambulatory boys with Duchenne muscular dystrophy (DMD) with a nonsense mutation, prior corticosteroid use, and stable baseline dosing; findings indicated that deflazacort was more effective in delaying the loss of physical milestones compared to prednisone/prednisolone in this specific population.