Viltolarsen
Viltolarsen
Viltolarsen (pronounced vil-toh-lar-sen) is a antisense oligonucleotide drug used 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.
Etymology
The name "Viltolarsen" is derived from the drug's chemical structure. The "vil" part of the name refers to the drug's antisense oligonucleotide nature, while "tolar" is a reference to the drug's ability to target and bind to specific sequences of RNA, and "sen" is a common suffix for drugs in this class.
Usage
Viltolarsen is used to treat Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact. This drug is specifically designed for patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Mechanism of Action
Viltolarsen works by binding to exon 53 of the dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing. Skipping exon 53 allows for the production of an internally truncated, but functional form of dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
Related Terms
External links
- Medical encyclopedia article on Viltolarsen
- Wikipedia's article - Viltolarsen
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