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Myotonic dystrophy type 1 can cause progressive muscle weakness, myotonia, gastrointestinal dysmotility, cardiac conduction abnormalities, and cataracts. This autosomal-dominant condition is caused by a repeat expansion in the myotonic dystrophy type 1 protein kinase (DMPK) gene, leading to toxic RNA and abnormal gene splicing; there is currently no cure. However, there have been recent advances in developing therapies that target the disease’s abnormal gene expansions.
Researchers now report phase 1–2 results from an ongoing industry-funded trial of the safety and pharmacodynamics of delpacibart etedesiran, an antibody–small interfering RNA (siRNA) conjugate designed to enhance siRNA delivery to cardiac and skeletal muscle and silenc…