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

•No medical intervention can completely arrest aneurysmal growth.•Drugs with immediate translational potential in Marfan syndrome include resveratrol, doxycycline, statins, indomethacin, and allopurinol.•They target nitric oxide signaling, extracellular matrix homeostasis, and transforming growth fa...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Drug discovery today 2024-07, Vol.29 (7), p.104023, Article 104023
Hauptverfasser: Van Den Heuvel, Lotte J.F., Peeters, Silke, Meester, Josephina A.N., Coucke, Paul J., Loeys, Bart L.
Format: Artikel
Sprache:eng
Schlagworte:
Online-Zugang:Volltext
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
Beschreibung
Zusammenfassung:•No medical intervention can completely arrest aneurysmal growth.•Drugs with immediate translational potential in Marfan syndrome include resveratrol, doxycycline, statins, indomethacin, and allopurinol.•They target nitric oxide signaling, extracellular matrix homeostasis, and transforming growth factor beta signaling.•Clinical trials are warranted to uncover the full potential of alternative drug targets. 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.
ISSN:1359-6446
1878-5832
1878-5832
DOI:10.1016/j.drudis.2024.104023