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viltolarsen

Viltolarsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients. It is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.