Preclinical development of antisense conjugates for the treatment of myotonic dystrophy 1

<p>Myotonic dystrophy type 1 (DM1) is a complex autosomal dominant neuromuscular disorder that affects approximately 1 in 8,000 individuals. DM1 impacts multiple organ systems, including skeletal muscle, heart and central nervous system (CNS), leading to a diverse range of symptoms. Despite on...