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These may be due to defective DNA synthesis inhibiting replication in a site with a high turnover of cells

Funding All the activities related to the preparation of this article have been supported by an unconditioned grant from Ipsen

Safety, tolerability, and pharmacokinetics of casimersen in patients with duchenne muscular dystrophy amenable to exon 45 skipping: a randomized, double-blind, placebo-controlled, dose-titration trial