In a previous study, a clear and highly significant difference was observed between (untreated) hDMDdel52/ mdx mice and C57BL6/J controls [ 35 ]. hDMDdel52/ mdx mice were analyzed at baseline (6 weeks) and after 8 and 13 weekly injections of 19.8 mg/kg AON-C19.
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Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy.
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