An exploration of alternative therapeutic targets for aortic disease in Marfan syndrome.

Drug Discov Today

Center for Medical Genetics Antwerp, University of Antwerp, Antwerp, Belgium; Antwerp University Hospital, Edegem, Belgium; Department of Human Genetics, Radboud University Medical Center, Nijmegen, the Netherlands. Electronic address:

Published: July 2024

Marfan syndrome is a rare connective tissue disorder that causes aortic dissection-related sudden death. Current conventional treatments, beta-blockers, and type 1 angiotensin II receptor blockers are prescribed to slow down aortic aneurysm progression and delay (prophylactic) aortic surgery. However, neither of these treatments ceases aortic growth completely. This review focuses on potential alternative therapeutic leads in the field, ranging from widely used medication with beneficial effects on the aorta to experimental inhibitors with the potential to stop aortic growth in Marfan syndrome. Clinical trials are warranted to uncover their full potential.

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Source
http://dx.doi.org/10.1016/j.drudis.2024.104023DOI Listing

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