Summary

This AI-generated content is provided without warranty, with no liability accepted for reliance on it. Learn more.

• This summary is based on the review of three randomized controlled trial(s). ^[1-3]
• Time to Stand from Supine (TTSTAND): Viltolarsen-treated patients demonstrated stabilization of TTSTAND over 109 weeks, with significant slowing of motor function decline over the 192-week term extension (LTE) study compared to the historical control group, which experienced decline. In the 24-week trial, TTSTAND improved in the viltolarsen group (-0.19 s) compared to controls (0.66 s).
• Time to Run/Walk 10 Meters and 6-Minute Walk Test (6MWT): The 24-week trial showed improved walking speed in viltolarsen-treated participants (0.23 m/s) versus controls (-0.04 m/s). Viltolarsen-treated participants also improved their 6MWT distance (+28.9 m) compared to a decline in controls (-65.3 m).
• Motor Function Stabilization: Viltolarsen treatment resulted in motor function stabilization, with significant differences from historical controls at several time points, supporting long-term benefits in slowing disease progression in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.
• Viltolarsen was well tolerated across all studies, with most treatment-emergent adverse events being mild or moderate, and no serious adverse events, deaths, or discontinuations reported. In the 192-week LTE and 24-week trials, there were no treatment-related serious adverse events, no participants discontinued due to adverse effects, and no dose reductions or interruptions were required.
• All studies included boys aged 4 to <10 years with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, demonstrating significant drug-induced dystrophin production (mean de novo dystrophin levels: 5.7%–5.9% of normal) across both low-dose and high-dose cohorts in the 24-week trial.