Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome.

@article{Gordon2012ClinicalTO,
  title={Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome.},
  author={Leslie B Gordon and Monica Kleinman and David T. Miller and Donna S Neuberg and Anita Giobbie-Hurder and Marie D. Gerhard-Herman and Leslie B. Smoot and Catherine M. Gordon and Robert H. Cleveland and Brian Scott Snyder and Brian J. Fligor and Walter Robert Bishop and Paul Statkevich and Amy Regen and Andrew L. Sonis and Susan I. Riley and Christine Ploski and Annette Correia and Nicolle L Quinn and Nicole N J Ullrich and Ara Nazarian and Marilyn G. Liang and Susanna Y. Huh and Armin Schwartzman and Mark W Kieran},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  year={2012},
  volume={109 41},
  pages={
          16666-71
        }
}
Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare, fatal, segmental premature aging syndrome caused by a mutation in LMNA that produces the farnesylated aberrant lamin A protein, progerin. This multisystem disorder causes failure to thrive and accelerated atherosclerosis leading to early death. Farnesyltransferase inhibitors have ameliorated disease phenotypes in preclinical studies. Twenty-five patients with HGPS received the farnesyltransferase inhibitor lonafarnib for a… CONTINUE READING
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