PathologyJournal of the American Heart AssociationPeer-reviewed
Fabry Cardiomyopathy: Myocardial Fibrosis, Inflammation and Down-Regulation of Mannose-6-Phosphate Receptors cause Low accessibility to Enzyme Replacement Therapy
Andrea Frustaci, Romina Verardo, Michele Magnocavallo…·Cristina Chimenti
This study investigates the limited effectiveness of enzyme replacement therapy (ERT) in advanced Fabry disease cardiomyopathy, identifying myocardial fibrosis, inflammation, and reduced mannose-6-phosphate receptors as barriers to therapy.