Association of lonafarnib treatment vs no treatment with mortality rate in patients with Hutchinson-Gilford progeria syndrome

LB Gordon, H Shappell, J Massaro, RB D'Agostino… - Jama, 2018 - jamanetwork.com
Importance Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare fatal
premature aging disease. There is no approved treatment. Objective To evaluate the …

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

LB Gordon, J Massaro, RB D'Agostino Sr… - Circulation, 2014 - Am Heart Assoc
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 …

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

LB Gordon, ME Kleinman, DT Miller… - Proceedings of the …, 2012 - National Acad Sciences
Hutchinson–Gilford progeria syndrome (HGPS) is an extremely rare, fatal, segmental
premature aging syndrome caused by a mutation in LMNA that produces the farnesylated …

Clinical trial of the protein farnesylation inhibitors lonafarnib, pravastatin, and zoledronic acid in children with Hutchinson-Gilford progeria syndrome

LB Gordon, ME Kleinman, J Massaro… - Circulation, 2016 - Am Heart Assoc
Background: Hutchinson-Gilford progeria syndrome is an extremely rare, fatal, segmental
premature aging syndrome caused by a mutation in LMNA yielding the farnesylated …

[HTML][HTML] Hutchinson-Gilford progeria syndrome

LB Gordon, WT Brown, FS Collins - 2019 - europepmc.org
Hutchinson-Gilford progeria syndrome (HGPS) is characterized by clinical features that
typically develop in childhood and resemble some features of accelerated aging. Children …

Disease progression in Hutchinson-Gilford progeria syndrome: impact on growth and development

LB Gordon, KM McCarten, A Giobbie-Hurder… - …, 2007 - publications.aap.org
OBJECTIVES. Hutchinson-Gilford progeria syndrome is a rare and uniformly fatal segmental
“premature aging” disease that affects a variety of organ systems. We sought to more clearly …

Lonafarnib: first approval

S Dhillon - Drugs, 2021 - Springer
Lonafarnib (Zokinvy™) is an orally active farnesyltransferase inhibitor developed by Eiger
BioPharmaceuticals under license from Merck & Co. for the treatment of hepatitis D virus …

Phenotype and course of Hutchinson–Gilford progeria syndrome

MA Merideth, LB Gordon, S Clauss… - New England journal …, 2008 - Mass Medical Soc
Background Hutchinson–Gilford progeria syndrome is a rare, sporadic, autosomal dominant
syndrome that involves premature aging, generally leading to death at approximately 13 …

Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome

M Puttaraju, M Jackson, S Klein, A Shilo, CF Bennett… - Nature medicine, 2021 - nature.com
Hutchinson–Gilford progeria syndrome (HGPS) is a rare, invariably fatal childhood
premature aging disorder caused by a pre-messenger RNA (mRNA) splicing defect in the …

Hutchinson–Gilford progeria syndrome

NJ Ullrich, LB Gordon - Handbook of clinical neurology, 2015 - Elsevier
Hutchinson–Gilford progeria syndrome (HGPS) is an extremely rare, uniformly fatal,
segmental “premature aging” disease in which children exhibit phenotypes that may give us …