Barth Syndrome Management Gets a Boost With Targeted Elamipretide Therapy
A comprehensive GeneReviews update on Barth syndrome outlines diagnosis, new targeted therapy, and surveillance strategies for this rare X-linked disorder.
Cardiovascular disease prevention, blood pressure, cholesterol, and heart function
518 articles
A comprehensive GeneReviews update on Barth syndrome outlines diagnosis, new targeted therapy, and surveillance strategies for this rare X-linked disorder.
The first disease-specific treatment for Barth syndrome gains FDA accelerated approval, targeting mitochondrial dysfunction to restore muscle strength.
Deleting GTPBP3 in endothelial cells blocks angiogenesis via mtROS overload — and a mitochondria-targeted antioxidant reverses the damage.
A 2-year clinical trial testing Juice Plus+ products against low-grade inflammation and biological aging markers in overweight seniors was terminated before completion.
Mendelian randomization links circulating inflammatory proteins to retinal microvascular changes, heart disease risk, and lifespan — establishing causal direction.
A 2025 review maps the molecular mechanisms driving cardiovascular aging—from inflammaging to ECM remodeling—and outlines emerging therapies.
A Phase 1/2 trial tests whether nicotinamide riboside can restore NAD+ balance and improve heart function in systolic HF patients.
A NYC cohort study finds social adversity dramatically raises mortality and rehospitalization in people living with HIV and heart failure.
A major trial shows evolocumab dramatically reduces cardiovascular events in stent patients who've never had a heart attack.
Researchers propose targeting lymph node entry mechanisms to improve immune tolerance in cardiac transplant recipients, potentially reducing rejection.
Cas13-based RNA editing shows promise for treating hereditary hypertrophic cardiomyopathy by correcting disease-causing mutations at the RNA level.
A Circulation review from Harvard proposes the 'target trial framework' to improve how observational data is used to assess cardiovascular device safety and effectiveness.