ns 065

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically binds exon 53 of the dystrophin mRNA precursor, restoring the amino acid open-reading frame through exon 53 skipping and resulting in the production of a shortened but functionally relevant dystrophin protein. Viltolarsen provides a mechanistically precise experimental agent for investigating exon-skipping therapies and molecular pathology in Duchenne muscular dystrophy research.