Evaluating the Therapeutic Potential of Del-Desiran in Myotonic Dystrophy Type 1: The HARBOR Trial

Myotonic dystrophy type 1 (DM1) is known as a progressive neuromuscular disorder that impacts multiple organ systems, including skeletal and smooth muscles, the heart, eyes, and both the endocrine and central nervous systems. The condition is caused by an expansion of CUG triplet repeats in the myotonic dystrophy protein kinase (DMPK) gene, with healthy individuals typically exhibiting fewer than 35 repeats. In contrast, those with DM1 may have thousands of these repeats, which contribute to the characteristic symptoms of the disease.¹