viltolarsen (Pending FDA Approval)

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Duchenne Muscular Dystrophy

Pending FDA approval for Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

Duchenne Muscular Dystrophy

Pending FDA approval for Duchenne muscular dystrophy (DMD) in children aged ≥3 years who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

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Pharmacology

Mechanism of Action

Morpholino antisense oligonucleotide designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping

Exon skipping is intended to allow for production of an internally truncated dystrophin protein

The underlying cause of Duchenne muscular dystrophy (DMD) is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.