How does Vyondys 53 work?

Vyondys 53 (golodirsen) is a prescription medication used to treat Duchenne muscular dystrophy (DMD) in patients with a specific genetic mutation amenable to exon 53 skipping.

Understanding Duchenne Muscular Dystrophy (DMD)

DMD is a genetic disorder caused by mutations in the dystrophin (DMD) gene, which encodes the dystrophin protein. This protein is crucial for maintaining muscle integrity. Individuals with DMD often have a deletion mutation affecting exons 43-55, leading to a lack of functional dystrophin. Without this protein, muscle tissue deteriorates, becoming replaced by fibrous tissue and fat, ultimately resulting in progressive muscle weakness.

Mechanism of Action

Vyondys 53 is an antisense oligonucleotide administered by intravenous infusion that works by binding to exon 53 of dystrophin pre-mRNA. This results in this exon being skipped over during mRNA processing. This exon skipping enables the production of a shortened but partially functional dystrophin protein, which may help slow disease progression and improve muscle function.

By promoting exon skipping, Vyondys 53 facilitates the production of a dystrophin protein that, while not fully functional, provides some protective benefits to muscle fibers. This approach does not cure DMD but aims to reduce muscle deterioration associated with the disease.