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. 2020 Sep 3;10(3):111. doi: 10.3390/jpm10030111

Table 2.

Comparison of FDA-approved exon-skipping therapies for Duchenne muscular dystrophy (DMD). Mean dystrophin protein production (as a percentage), relative to healthy controls, is presented based on Western blot data. Baseline values are included for reference.

Therapy Baseline
(% of Normal)
Dystrophin Production
(% of Normal)
Side Effects
Eteplirsen [44] 0.08 0.93 No severe or moderate adverse events
8 mild events considered related to treatment [45]
Golodirsen [46] 0.095 1.019 2 moderate adverse events (infection and pyrexia)
8 mild events considered related to treatment
Viltolarsen [43] 0.3 (dose of 40 mg/kg)
0.6 (dose of 80 mg/kg)
5.7 (dose of 40 mg/kg)
5.9 (dose of 80 mg/kg)
No severe or moderate adverse events
No mild events considered related to treatment