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Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review

Waldrop MA, Yaou RB, Lucas KK, et al. Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review. Journal of neuromuscular diseases 2020;7:217-229   Abstract BACKGROUND: Eteplirsen, the first FDA-approved RNA-modifying therapy for DMD, is applicable to approximately 13% of patients with DMD. Because multiple exonic deletions are amenable to exon 51 skipping, […]