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Brand: Viltepso
Viltolarsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. It is a targeted therapy designed to specifically bind to exon 53 of the DMD pre-mRNA, leading to its exclusion during mRNA processing. This exon skipping allows for the production of a partially functional dystrophin protein.
Viltolarsen is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.
Outcome:
Mechanism:
Most likely new formulation: Subcutaneous administration (Year: 2025, 50% confidence)
Based on current clinical trial data and FDA approval, viltolarsen is likely to remain a prescribed treatment for DMD amenable to exon 53 skipping.
Antisense Oligonucleotide
Phosphorodiamidate morpholino oligomer (PMO)