— Disease indication: metabolic disorders including insulin resistance and diabetic nephropathy
Drug format: chemically modified synthetic microRNA (miRNA)
Drug class: first-in-class for metabolic disease
Research stage and Preliminary data:
Target: miR-29 mimics can increase the gene expressio…
— Technology Description
Dr. Fumihiko Urano at Washington University in St. Louis (WUSTL) and colleagues have developed therapeutic strategies to treat Wolfram syndrome. Wolfram syndrome is a rare, life-threating, genetic disorder characterized by insulin-dependent diabetes mellitus, diabetes insipi…
— Lysosomal storage disorders result in severe, multi-system dysfunction and frequently, early death. Currently, a number of lysosomal storage disorders are treated with enzyme replacement therapy (ERT), which requires periodic infusions of the deficient enzyme. However, in a number of instances the…
— Background: About 422 million people worldwide and over 29 million Americans have diabetes. The prevalence of diabetes is increasing and an important cause of blindness, amputation, kidney failure, and one of the leading causes of premature death. Diabetes is associated with significant costs. In th…
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