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FREEDOM-DM1: Nonclinical Data Support the Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 in Adults with Myotonic Dystrophy Type 1 (DM1)
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Nonclinical Data Demonstrate the Potential of the Enhanced Delivery Oligonucleotide (EDO) PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy
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PGN-EDODM1: Single- and Repeat-Dose Nonclinical Data Indicated Mechanistic and Meaningful Activity for Potential Treatment of Myotonic Dystrophy Type 1 (DM1)