For research use only. Not for therapeutic Use.
Viltolarsen is an antisense oligonucleotide medication developed by Nippon Shinyaku Co., Ltd., for treating Duchenne muscular dystrophy (DMD). It is designed to skip exon 53 of the dystrophin gene, enabling the production of a truncated but functional form of the dystrophin protein. This approach is targeted at patients with DMD who have genetic mutations amenable to exon 53 skipping. Viltolarsen received accelerated approval in Japan in 2019 for treating DMD in patients with confirmed exon 53 skipping mutations.
| Catalog Number | I048035 |
| CAS Number | 2055732-84-6 |
| Synonyms | NS-065/NCNP-01 |
| Molecular Formula | C244H381N113O88P20 |
| Purity | 98% |
| Appearance | Solid |
