Neurologic features of Hutchinson-Gilford progeria syndrome after lonafarnib treatment.

ABSTRACT:

Objectives

The objective of this study was to retrospectively evaluate neurologic status pre- and posttreatment with the oral farnesyltransferase inhibitor lonafarnib in children with Hutchinson-Gilford progeria syndrome (HGPS), a rare, fatal disorder of segmental premature aging that results in early death by myocardial infarction or stroke.

Methods

The primary outcome measure for intervention with lonafarnib was to assess increase over pretherapy in estimated annual rate of weight gain. In this study, neurologic signs and symptoms were compared pre- and posttreatment with lonafarnib.

Results

Twenty-six participants were treated for a minimum of 2 years. Frequency of clinical strokes, headaches, and seizures was reduced from pretrial rates. Three patients with a hist

SUBMITTER: Ullrich NJ 

PROVIDER: S-EPMC3776537 | biostudies-literature | 2013 Jul

REPOSITORIES: biostudies-literature