Treatment advances for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe X-linked muscle wasting disease with childhood onset that leads to loss of ambulation in the teenage years and mortality in the second or third decade of life. Prior to 10 years ago, disease-modifying pharmacologic intervention was limited to glucocorticoid steroids. However, in the past decade, several new medications have been approved for use in DMD. These medications work by different mechanisms along the disease pathway that lead to progressive muscle damage in DMD.

Seven new medications for DMD have been approved for use by the United States Food and Drug Administration since 2016. These include vamorolone (AGAMREE), which is a novel dissociative glucocorticoid; eteplirsen (EXONDYS 51), golodirsen (VYONDYS 53), vitolarsen (VILTEPSO) and casimersen (AMONDYS-45), which are the so-called 'exon skipping' phosphorodiamidate morpholino oligomers or antisense oligonucleotides; delandistrogene moxeparvovec (ELEVIDYS), which is a microdystrophin gene transfer therapy; and givinostat (DUVYZAT), which is a histone deacetylase inhibitor.

This review summarizes the mechanism of action, key safety considerations and available evidence on motor function impact that these novel medications have demonstrated in DMD.