Golodirsen (Vyondys 53) is a medication used to treat Duchenne muscular dystrophy (DMD) in adults and children. It is a type of antisense oligonucleotide (ASO) that works by targeting and altering the genetic code of a protein called dystrophin. By doing so, it helps to increase the amount of dystrophin in the muscles of those with DMD. Golodirsen has been approved by the Food and Drug Administration (FDA) to treat DMD in adults and children aged 6 years and older. The most common side effects of Golodirsen are nausea, headache, injection site reactions, and increases in liver enzymes.
Golodirsen, sold under the brand name Vyondys 53, is a medication used for the treatment of Duchenne muscular dystrophy (DMD). It is a first-in-class antisense oligonucleotide medication designed to treat DMD by inducing exon skipping. Exons are sections of genes that code for proteins. In DMD, mutations in the dystrophin gene can cause some exons to be not included when the protein is made. This can lead to a protein that is not functional, which causes muscle weakness. Golodirsen is designed to bind to pre-mRNA (messenger RNA precursor), a molecule involved in protein production, and tell the cell to skip exon 53 during the splicing process. This can allow the cell to produce a partially functional dystrophin protein, which can help to improve muscle function.
Golodirsen is approved for use in people with DMD who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is given as an intravenous infusion once weekly. The most common side effects of golodirsen include upper respiratory infection, headache, fever, inflammation of the injection site, and vomiting.
Golodirsen is a relatively new medication, and it is still being studied to determine its long-term effects. However, it has been shown to be effective in improving muscle function in people with DMD.