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FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1
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CONNECT1-EDO51 and CONNECT2-EDO51: Phase 2 Study Designs to Evaluate Safety and Efficacy for Duchenne Muscular Dystrophy 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
