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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Dades bibliogràfiques
Publicat a:Circulation
Autors principals: 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
Idioma:Inglês
Publicat: 2016
Matèries:
Article
Accés en línia: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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