Briefly Noted

Improving the Outcome for Patients with Marfan Syndrome

Marfan syndrome (MFS) is an autosomal dominant genetic disorder caused by mutations in FBN1, which encodes fibrillin 1. Beyond the defects in connective tissues, the major cause of morbidity and mortality in patients with this condition is aortic root dilatation, and MFS pathogenesis has been demonstrated to involve disordered transforming growth factor-β (TGF-β) activation and signaling. Investigators studied the effect of beta-blockers and the angiotensin-receptor blocker losartan in a mouse model of MFS and in human patients1^,2. Losartan decreased the occurrence of aortic root dilatation in the mouse model, and in humans, TGF-β concentrations were significantly lower in losartan or b…