NS 050/NCNP 03
Alternative Names: Exon 50 skipping antisense oligonucleotide; NS-050/NCNP-03Latest Information Update: 30 Jun 2023
At a glance
- Originator Nippon Shinyaku
- Developer National Center of Neurology and Psychiatry; Nippon Shinyaku
- Class Antisense oligonucleotides; Morpholines
- Mechanism of Action Dystrophin expression stimulants; RNA interference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
- Available For Licensing Yes
Highest Development Phases
- Preclinical Duchenne muscular dystrophy