Summary

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• Amondys 45 (casimersen) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.
• This summary is based on the review of one randomized controlled trial(s). ^[1]
• The study does not provide direct data or results related to the effectiveness of casimersen (amondys_45) in treating Duchenne muscular dystrophy (DMD); it focuses solely on safety and pharmacokinetics.
• No effectiveness outcomes or comparative effectiveness data with other drugs are discussed in the study.
• Treatment-emergent adverse events (TEAEs) occurred in all participants, with over 91.4% being mild and unrelated to casimersen; no serious adverse events, deaths, or dose reductions were reported, and no abnormalities in laboratory parameters or vital signs were attributed to the drug.
• Casimersen was well tolerated across participants aged 7-21 years with Duchenne muscular dystrophy amenable to exon 45 skipping, regardless of mobility status, with no specific safety concerns identified for particular population subgroups.
• Participants aged 7-21 years, including both limited ambulation and nonambulatory individuals with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping, demonstrated general tolerance to casimersen with mild, nonserious treatment-emergent adverse events (TEAEs) unrelated to the drug and consistent pharmacokinetic parameters across all dose levels.