Seeking a Cure for One of the Rarest Diseases: Progeria
- PMID: 27400897
- PMCID: PMC5101939
- DOI: 10.1161/CIRCULATIONAHA.116.022965
Seeking a Cure for One of the Rarest Diseases: Progeria
Keywords: Editorials; everolimus; lamin type A; lonafarnib; morpholinos; progeria; rare diseases; translational medical research.
Comment on
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Clinical Trial of the Protein Farnesylation Inhibitors Lonafarnib, Pravastatin, and Zoledronic Acid in Children With Hutchinson-Gilford Progeria Syndrome.Circulation. 2016 Jul 12;134(2):114-25. doi: 10.1161/CIRCULATIONAHA.116.022188. Circulation. 2016. PMID: 27400896 Free PMC article. Clinical Trial.
References
-
- Online. Mendelian Inheritance in Man. OMIM Gene Map Statistics. [Accessed May 19, 2016]. http://www.omim.org/statistics/geneMap.
-
- National Institutes of Health. About NCATS, National Center for Advancing Translational Sciences. [Accessed May 19, 2016]. https://ncats.nih.gov/about.
-
- Gordon LB, Kleinman ME, Massaro J, D’Agostino RB, Shappell H, Gerhard-Herman M, Smoot LB, Gordon CM, Cleveland RH, Nazarian A, Snyder BD, Ullrich NJ, Silvera VM, Liang MG, Quinn N, Miller DT, Huh SY, Dowton AA, Littlefield K, Greer MM, Kieran MW. Clinical trial of the protein farnesylation inhibitors lonafarnib, pravastatin and zoledronic acid in children with Hutchinson-Gilford Progeria Syndrome. Circulation. 2016;134:114–125. doi: 10.1161/CIRCULATIONAHA.116.022188. - DOI - PMC - PubMed
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- Gordon LB, Brown WT, Collins FS. Hutchinson-Gilford progeria syndrome. In: Pagon RA, Adam MP, Ardinger HH, Wallace SE, Amemiya A, Bean LJH, Bird TD, Fong CT, Mefford HC, Smith RJH, Stephens K, editors. GeneReviews® [Internet] Seattle, WA: University of Washington, Seattle; 1993–2016. 2003, updated 2015. - PubMed
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