Article
Impact of farnesylation inhibitors on survival in Hutchinson-Gilford progeria syndrome.
Circulation - 1 Jul 2014
Gordon Leslie B, Massaro Joe, D'Agostino Ralph B, Campbell Susan E, Brazier Joan, Brown W Ted, Kleinman Monica E, Kieran Mark W
Abstract excerpt
BACKGROUND: Hutchinson-Gilford progeria syndrome is an ultrarare segmental premature aging disease resulting in early death from heart attack or stroke. There is no approved treatment, but starting in 2007, several recent single-arm clinical trials administered inhibitors of protein farnesylation aimed at reducing toxicity of the disease-producing protein progerin. No study assessed whether treatments influence...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.