Article
Survey of plasma proteins in children with progeria pre-therapy and on-therapy with lonafarnib.
Pediatric research - 1 May 2018
Gordon Leslie B, Campbell Susan E, Massaro Joseph M, D'Agostino Ralph B, Kleinman Monica E, Kieran Mark W, Moses Marsha A
Abstract excerpt
BackgroundHutchinson-Gilford progeria syndrome (HGPS) is an ultra-rare, fatal, segmental premature aging syndrome caused by the aberrant lamin A protein, progerin. The protein farnesyltransferase inhibitor, lonafarnib, ameliorates some aspects of cardiovascular and bone disease.MethodsWe performed a prospective longitudinal survey of plasma proteins in 24 children with HGPS (an estimated 10% of the world's...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
