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Review
. 2024 Jul;29(7):104023.
doi: 10.1016/j.drudis.2024.104023. Epub 2024 May 13.

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

Affiliations
Review

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

Lotte J F Van Den Heuvel et al. Drug Discov Today. 2024 Jul.

Abstract

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.

Keywords: Marfan syndrome; aortic aneurysm; aortic biomarkers; aortic dissection; therapy.

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