Viltolarsen: First Approval

@article{Dhillon2020ViltolarsenFA,
  title={Viltolarsen: First Approval},
  author={Sohita Dhillon},
  journal={Drugs},
  year={2020},
  volume={80},
  pages={1027-1031}
}
Viltolarsen (Viltepso ® 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 the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional… 

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CPAA_A_288842 235..242

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