Which medications in the drug class Morpholino Antisense Oligomers, Neurology are used in the treatment of Muscular Dystrophy?

Updated: Aug 17, 2020
  • Author: Twee T Do, MD; Chief Editor: Jeffrey D Thomson, MD  more...
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Answer

Morpholino Antisense Oligomers, Neurology

These agents bind to various exons (eg, 45, 51, 53) of dystrophin pre-mRNA, resulting in exclusion of the particular exon (ie, skipping) during mRNA processing. Exon skipping is intended to allow production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to specific exon skipping.

Eteplirsen (Exondys 51)

Eteplirsen is a phosphorodiamidate morphino oligomer (PMO), the first of its class. It is indicated for Duchenne MD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. It is administered as a once-weekly IV infusion.

Golodirsen (Vyondys 53)

A second PMO was approved for Duchenne MD in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. 

Viltolarsen (Viltepso)

Antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass that binds to exon 53 of dystrophin pre-mRNA. This results in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. It is indicated for Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation that is amenable to exon 53 skipping.

Casimersen (Amondys 45)

Casimersen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMO binds to exon 45 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Casimersen is indicated for Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping. 


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