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FREEDOM-DM1: Final Results from a Phase 1, Placebo-controlled SAD Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1)
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Previous:
Development of PGN-EDODM1, a Novel Enhanced Delivery Oligonucleotide that Targets the Root Cause of Myotonic Dystrophy Type 1
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The FREEDOM-DM1 clinical trial demonstrated strong splicing correction with single doses of PGN-EDODM1, with an acceptable safety profile
