
Pipeline
We are advancing and expanding our innovative AOC pipeline to offer treatment options for patients and their families across a wide range of therapeutic areas. We have three AOC programs for three distinct rare disease in clinical development from our muscle disease franchise: myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne Muscular Dystrophy (DMD). We are also advancing AOCs for additional DMD programs, rare skeletal muscle, rare cardiac program, and continue to broaden our reach of AOCs to other indications like cardiology and immunology through internal discovery & partnerships.