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Description

Frontiers in Endocrinology, 14, 1244432

Approved ASO drugs target exon 45 (casimersen), 264,496 exon 51 (eteplirsen), 497,498 and exon 53 (golodirsen and viltolarsen) 80,263 to induce specific exon skipping in the dystrophin gene, thus delaying disease progression