An analysis of more than 1,000 small molecules has identified dozens of compounds that could be effective to treat Marfan syndrome (MFS), an inherited disorder affecting connective tissue, primarily in the heart and blood vessels, the skeleton, and the eyes. In particular, the researchers from Cambridge University found that glycogen synthase kinase-3β (GSK-3β) could be a target to develop new therapies based on its inhibition.

Researchers from Shanghai Institute of Materia Medica and affiliated organizations recently reported the synthesis and evaluation of novel α-pyrone III derivatives as potential therapeutic agents for the treatment of atherosclerosis.

Rocket Pharmaceuticals Inc. has added RP-A601 to its cardiac gene therapy portfolio for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM).

Severe toxicities associated with immune checkpoint inhibitor (ICI) therapy are a major challenge of this anticancer therapy approach. While myocarditis is a rare immune-related adverse event in patients receiving ICIs, it has a nearly 50% mortality rate and its pathogenesis is poorly understood. In the current study, researchers from Vanderbilt University and affiliated organizations published data from a study that evaluated a novel mouse model recapitulating clinical and pathological features of ICI-associated myocarditis (ICI-MC).

Ischemic stroke comprises about 87% of stroke incidence. The nuclear factor κ B (NF-κB) cascade is a major regulatory pathway of inflammation and is activated in response to ischemic stroke in humans and in rodents during middle cerebral artery occlusion (MCAO).

The FDA has awarded orphan drug designation to Tenaya Therapeutics Inc.'s gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC). TN-401 is an adeno-associated virus (AAV)-based gene therapy being developed for the treatment of genetic ARVC caused by plakophilin-2 (PKP2) gene mutations.

Noncoding RNAs, such as long noncoding RNAs (lncRNAs), are important regulators and markers of cardiovascular diseases. Saphenous veins are frequently used in coronary artery bypass surgery, but about 50% of the saphenous vein grafts fail in the first 10 years after surgery due to neointima formation.

Recent findings have suggested glycosylated apolipoprotein J (ApoJ-Glyc) levels to be a marker for the diagnosis of myocardial ischemia. Analysis of this marker was performed in a cohort of patients with chest pain suggestive of acute coronary syndrome (ACS) (N=404). ApoJ-Glyc serum levels were analyzed with a novel ELISA assay that targets a specific glycosylated variant of ApoJ (ApoJ-GlycA6). It was found that 291 patients were diagnosed as having a nonischemic event, while 113 were classified as having an ischemic event, 33 as STEMI, 48 as non-STEMI, 27 as unstable angina pectoris and 5 as unclassifiable ACS patients.