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viltolarsen
- Development stage
- Approved
- Lead developer
- Nippon Shinyaku
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
Viltolarsen is an antisense phosphorodiamidate morpholino oligonucleotide (PMO) designed for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 53 skipping. It works by binding to exon 53 of the DMD pre-mRNA, causing this exon to be skipped during mRNA splicing. This results in the production of a truncated but partially functional dystrophin protein, which helps stabilize muscle cell membranes and slow disease progression. Viltolarsen is administered as an intravenous infusion and was developed through collaboration between Nippon Shinyaku and Japan’s National Center of Neurology and Psychiatry. It received FDA accelerated approval in August 2020 for use in patients with DMD who have mutations suitable for exon 53 skipping, representing about 8% of all DMD cases[1][3][5][6][8].
- Brand names
- Viltepso
- Other names
- viltolarsen
02
Targets
Neuraminidase
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