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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Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha: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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