Cardiac abnormalities in patients with Hutchinson-Gilford progeria syndrome
A Prakash, LB Gordon, ME Kleinman… - JAMA …, 2018 - jamanetwork.com
Importance Hutchinson-Gilford progeria syndrome (HGPS) is an ultrarare disorder
associated with premature death due to cardiovascular events during the second decade of
life. However, because of its rarity (107 identified living patients), the natural history of
cardiac disease remains uncharacterized. Therefore, meaningful cardiac end points for
clinical trials have been difficult to establish. Objective To examine the course of appearance
of cardiac abnormalities in patients with HGPS to identify meaningful cardiac end points for …
associated with premature death due to cardiovascular events during the second decade of
life. However, because of its rarity (107 identified living patients), the natural history of
cardiac disease remains uncharacterized. Therefore, meaningful cardiac end points for
clinical trials have been difficult to establish. Objective To examine the course of appearance
of cardiac abnormalities in patients with HGPS to identify meaningful cardiac end points for …