exon 53 of dystrophin pre-mRNA — Drug Target

All drugs that target exon 53 of dystrophin pre-mRNA — marketed and clinical-stage. Includes 1 drug classes acting on this target.

Drug classes

Antisense Oligonucleotide [EPC]

Marketed (3)

• Vyondys 53 · Sarepta · Antisense Oligonucleotide [EPC] · Rare Disease
  Vyondys 53 works by binding to specific RNA sequences to prevent the production of a faulty dystrophin protein.
• Amondys 45 · Sarepta · Antisense Oligonucleotide [EPC] · Rare Disease
  Amondys 45 works by binding to specific RNA sequences to prevent the production of a faulty protein that causes Duchenne muscular dystrophy.
• Viltepso · Nippon Shinyaku · Rare Disease
  Viltepso works by binding to exon 53 of dystrophin pre-mRNA, allowing for the production of a partially functional dystrophin protein.