PGN-EDODM1 Nonclinical Data Demonstrate Potential for Meaningful Impact in Myotonic Dystrophy Type 1 (DM1): Support for Phase 1 Clinical Trial Design

Holland A, Klein A, Godfrey C, Larkindale J, Svenstrup N, Bracegirdle S, Furling D, Garg B, McArthur J, Foy J, Mellion M, Goyal J

Molecular therapy