Viltolarsen: Side Effects, Uses & Monitoring

Labeled uses

VILTEPSO 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 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO [see Clinical Studies (14 )]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.