In honor of Muscular Dystrophy Awareness Month, held annually in September, NeurologyLive® looks back at the decade-long expansion of the DMD treatment landscape and the data that supports each approval.
News|Articles|March 26, 2025
Evaluating the Therapeutic Potential of Del-Desiran in Myotonic Dystrophy Type 1: The HARBOR Trial
Author(s)Isabella Ciccone, MPH
Fact checked by: Marco Meglio
The HARBOR study is an ongoing phase 3 global study focused on assessing del‑desiran, formerly known as AOC 1001, which aims to treat the underlying cause of DM1.
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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
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