Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
At the American Heart Association’s Scientific Sessions, E-Therapeutics plc presented preclinical data for ETX-258, a Galomic siRNA being developed for the treatment of heart failure.
Researchers from Ohio State University hypothesized that 12,13-diHOME may act by inhibiting the proarrhythmic molecule Ca2+/calmodulin-dependent kinase II (CaMKII).
An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.
Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiovascular disorder with around 85% of people with HCM remaining undiagnosed. There are no treatments approved for nonobstructive HCM (nHCM) to date.
Avidity Biosciences Inc. has announced two new precision cardiology development candidates targeting rare genetic cardiomyopathies. AOC-1086 targets phospholamban (PLN) cardiomyopathy and AOC-1072 targets protein kinase AMP-activated noncatalytic subunit γ2 (PRKAG2) syndrome.
Shenzhen Salubris Pharmaceuticals Co. Ltd. has described tertiary amine compounds reported to be useful for the treatment of stroke, atherosclerosis, thrombosis, coronary heart disease and aortic valve stenosis.
While the size of the market is enormous, drug development and treatments for women’s health care still lag behind what is offered for men. There has been a renaissance in the past few years, however, led by investors and companies that have wrestled with determining exactly what encompasses women’s health and how to meet its challenges.
Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a systemic autoimmune disease characterized by the inflammation of blood vessels, and it has been demonstrated that myeloperoxidase (MPO) is one of the major disease drivers of AAV, as it is a neutrophil surface protein that binds to ANCA.
The PLN-R14del mutation is often tied to dilated or arrhythmogenic cardiomyopathy, which can progress to end-stage heart failure. Cardiac phospholamban (PLN) plays a crucial role in calcium modulation in cardiomyocytes, which is disrupted by the PLN-R14del mutation.