Abstract
ViltepsoⓇ（viltolarsen） is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy （DMD）. It was discovered through joint research between Nippon Shinyaku Co., Ltd., and the National Center of Neurology and Psychiatry （NCNP）. It is an antisense oligonucleotide using phosphorodiamidate morpholino oligomer （PMO） that restores dystrophin protein lacking in DMD patients amenable to exon 53 skipping. VILTEPSO received marketing authorization under an accelerated approval pathway in Japan in March 2020. In the United States, it received accelerated approval from the US Food and Drug Administration （FDA） in August 2020.
