Study Design of AOC 1001-CS1, a Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA™) 

Antibody-Oligonucleotide Conjugates (AOCs) Demonstrate Potent and Durable Exon Skipping and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy

Prevalence of healthcare conditions and services used by patients with myotonic dystrophy (DM1 and DM2) prior to diagnosis; a real-world data analysis

Understanding the patients’ journey to the diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD): a real-world retrospective data analysis

DUX4 siRNA Optimization for the Development of an Antibody-Oligonucleotide Conjugate (AOC™) for the Treatment of FSHD