Clinical Trial of Protein Farnesylation Inhibitors Lonafarnib, Pravastatin and Zoledronic Acid in Children with Hutchinson-Gilford Progeria Syndrome

BACKGROUND: Hutchinson-Gilford progeria syndrome is an extremely rare, fatal, segmental premature aging syndrome caused by a mutation in LMNA yielding the farnesylated aberrant protein, progerin. Without progerin-specific treatment, death occurs at an average age of 14.6 years from an accelerated at...

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Udgivet i:Circulation
Main Authors: Gordon, Leslie B., Kleinman, Monica E, Massaro, Joe, D’Agostino, Ralph B., Shappell, Heather, Gerhard-Herman, Marie, Smoot, Leslie B., Gordon, Catherine M., Cleveland, Robert H., Nazarian, Ara, Snyder, Brian D., Ullrich, Nicole J., Silvera, V. Michelle, Liang, Marilyn G., Quinn, Nicolle, Miller, David T., Huh, Susanna Y., Dowton, Anne A., Littlefield, Kelly, Greer, Maya M., Kieran, Mark W.
Format: Artigo
Sprog:Inglês
Udgivet: 2016
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4943677/
https://ncbi.nlm.nih.gov/pubmed/27400896
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1161/CIRCULATIONAHA.116.022188
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