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Duchenne muscular dystrophy (DMD) is a devastating sex-linked genetic disorder that affects about 1 out of 3500 to 5000 males, causing progressive muscle weakness and impairment of cardiac and pulmonary function, eventually resulting in death. Various mutations in the gene for dystrophin prevent dystrophin production, resulting in necrosis of muscle cells. When the mutation disrupts the reading frame, a strategy to skip the abnormal pre-mRNA may restore the reading frame, producing a functional but truncated protein. Eteplirsen is a morpholino-based antisense oligonucleotide, designed to skip exon 51; a mutation “hot spot” in this vicinity results in 14% of all DMD mutations being amenable to frame correction by skipping exon 51 (Hum Mutat …