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FREEDOM-DM1: A Phase 1, Placebo-Controlled Single Ascending Dose Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1)
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Mechanistic Characterization of Enhanced Delivery Oligonucleotide (EDO) Platform
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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