2024 Muscular Dystrophy Association (MDA) Conference March 3 - March 6, 2024 Supporting Materials PGN-EDODM1: Single- and Repeat-Dose Nonclinical Data Indicated Mechanistic and Meaningful Activity for Potential Treatment of Myotonic Dystrophy Type 1 (DM1) 2.3 MB PGN-EDO51: Single- and Repeat-Dose Nonclinical Data Demonstrated Potential for the Treatment of Duchenne Muscular Dystrophy (DMD) 2.4 MB FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1 794.5 KB CONNECT1-EDO51 and CONNECT2-EDO51: Phase 2 Study Designs to Evaluate Safety and Efficacy for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping 858.3 KB
PGN-EDODM1: Single- and Repeat-Dose Nonclinical Data Indicated Mechanistic and Meaningful Activity for Potential Treatment of Myotonic Dystrophy Type 1 (DM1) 2.3 MB
PGN-EDO51: Single- and Repeat-Dose Nonclinical Data Demonstrated Potential for the Treatment of Duchenne Muscular Dystrophy (DMD) 2.4 MB
FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1 794.5 KB
CONNECT1-EDO51 and CONNECT2-EDO51: Phase 2 Study Designs to Evaluate Safety and Efficacy for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping 858.3 KB
