Cardiac Abnormalities in Patients With Hutchinson-Gilford Progeria Syndrome.

ABSTRACT:

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 use in future clinical trials.

Design, setting, and participants

In this prospective, cross-sectional, observational study, 27 consecutive patients with clinically and genetically confirmed classic HGPS were evaluated at a single center for

SUBMITTER: Prakash A 

PROVIDER: S-EPMC5875332 | biostudies-literature | 2018 Apr

REPOSITORIES: biostudies-literature