Impact of farnesylation inhibitors on survival in Hutchinson-Gilford progeria syndrome.

ABSTRACT:

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 patient survival. The key elements necessary for this analysis are a robust natural history of survival and comparison with a sufficiently large patient population that has been treated for a sufficient time period with disease-targeting medications.

Methods and results

We generated Kaplan-Meier survival analyses for the largest untreated Hutchinson-Gilford progeria sy

SUBMITTER: Gordon LB 

PROVIDER: S-EPMC4082404 | biostudies-literature | 2014 Jul

REPOSITORIES: biostudies-literature