Muscular Dystrophy Medication

Updated: Jul 01, 2021
  • Author: Twee T Do, MD; Chief Editor: Jeffrey D Thomson, MD  more...
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Medication

Medication Summary

Several agents have shown promise in management of Duchenne muscular dystrophy (MD). The mainstay of therapy has been the steroids; however, the lack of prolonged efficacy and the attendant adverse effects of higher-dose steroids limit their use.

Deflazacort, an oxazoline derivative of prednisolone, may offer therapeutic efficacy with fewer adverse effects.

The approval of eteplirsen, the first phosphorodiamidate morphino oligomer (PMO), provides an addition to the therapeutic armamentarium; however, continued approval of eteplirsen is contingent on postmarketing performance data. A second such agent, golodirsen, was subsequently approved. Other morpholino antisense oligomers, viltolarsen and casimersen, have also been approved by the FDA for use in the United States. [37, 38, 39, 40]   

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Corticosteroids

Class Summary

Glucocorticoid drugs delay the cytotoxic damage of MD to the necrosing muscle cells.

Prednisone (Deltasone, Prednisone Intensol, Rayos)

Prednisone exhibits beneficial effects in treatment of Duchenne MD in dosages ranging from 0.75 mg/kg/day to 1.5 mg/kg/day. Therapeutic effects are believed to be mediated through suppression of cytotoxic T-cell expression from necrotic muscles. Response to therapy may not last beyond several months, and adverse effects (eg, rapid weight gain, osteoporosis, myopathy, and growth retardation) limit its use.

Deflazacort (Emflaza)

Deflazacort, an oxazoline derivative of prednisolone, represents one of the newer corticosteroids for Duchenne MD. The FDA-approved dose is 0.9 mg/kg/day. It elicits a therapeutic response similar to that of prednisone.

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Morpholino Antisense Oligomers, Neurology

Class Summary

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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