Summary

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• Viltolarsen (Viltepso) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 53 skipping. It significantly increases dystrophin levels, stabilizing and even improving motor functions such as time to stand from supine, time to run/walk 10 meters, and performance on the 6-minute walk test.
• The information is derived from three randomized controlled trials, consistently reporting these findings about viltolarsen's effectiveness.
• Compared with historical controls from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS), patients treated with viltolarsen showed significant functional improvements or slower disease progression rates, suggesting its potential to alter DMD's typical trajectory among those amenable to exon 53 skipping therapy.
• Across all studies, viltolarsen demonstrated high safety and tolerability with most adverse events classified as mild or moderate. There were no reports of serious adverse events directly related to the treatment nor any discontinuations due to side effects; this favorable profile makes it suitable for the long-term use necessary in treating DMD.
• The primary population focus was boys aged between four and less than ten years at baseline, reflecting a pediatric group suffering from DMD treatable by exon 53 skipping therapy. However, further research would benefit older populations or those at advanced stages of the disease where efficacy remains uncertain.
• While direct comparisons with other treatments were not provided within these studies, based on the reported efficacy and safety profiles alone, it can be inferred that viltolarsen may serve as an important option, especially when considering subsets of patients whose mutations are amenable to exon 53 skipping therapies; the absence of severe side effects positions it favorably against others potentially having more concerning safety issues.