How does Vyondys 53 work?

Vyondys 53 (golodirsen) is a medication used to treat Duchenne muscular dystrophy (DMD) in patients who have a mutation of the DMD gene that is amenable to exon 53 skipping.

The DMD or dystrophin gene encodes an essential protein called dystrophin, which is needed for muscle integrity. People with DMD usually have a deletion mutation in exons 43-55. This means that their version of the dystrophin gene is missing some of the genetic material that they need to make a functional dystrophin protein.

Muscles lacking functional dystrophin protein get damaged when they contract. Without properly functioning dystrophin protein, normal muscle tissue is replaced by fibrous tissue and fat.

Vyondys 53 is an antisense oligonucleotide administered by intravenous infusion that works by binding to exon 53 of dystrophin pre-mRNA, which results in this exon being skipped over during mRNA processing. Skipping of exon 53 allows cells to join different exons together to produce a dystrophin protein that is shorter but has some function.