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Advancing the Use of Peptide-Conjugated Oligonucleotides to Target Neuromuscular Disorders: Enhanced Delivery Oligonucleotides for DMD and DM1
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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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Evaluation of PGN-EDODM1: FREEDOM-DM1 and FREEDOM2-DM1 Clinical Trials in Myotonic Dystrophy Type 1
