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CONNECT2-EDO51: A Phase 2 placebo-controlled study to evaluate PGNEDO51 safety and efficacy in people with Duchenne amenable to exon 51 skipping
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CONNECT1 – EDO51: A 12-week open-label Phase 2 study to evaluate PGN-EDO51 safety and efficacy in people with Duchenne amenable to exon 51 skipping
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Advancing the Use of Peptide-Conjugated Oligonucleotides to Target Neuromuscular Disorders: Enhanced Delivery Oligonucleotides for DMD and DM1