Viltolarsen: First Approval

Dhillon S. Viltolarsen: First Approval. Drugs 2020;80:1027-1031   Abstract Viltolarsen (Viltepso((R)) in Japan) is a phosphorodiamidate morpholino antisense oligonucleotide being developed by Nippon Shinyaku, in collaboration with the National Center of Neurology and Psychiatry (NCNP), for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores […]