Survey of plasma proteins in children with progeria pre-therapy and on-therapy with lonafarnib.
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Abstract |
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Hutchinson-Gilford progeria syndrome (HGPS) is an ultra-rare, fatal, segmental premature aging syndrome caused by the aberrant lamin A protein, progerin. The protein farnesyltransferase inhibitor, lonafarnib, ameliorates some aspects of cardiovascular and bone disease. |
Year of Publication |
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2018
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Journal |
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Pediatric research
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Date Published |
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2018
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ISSN Number |
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0031-3998
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URL |
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http://dx.doi.org/10.1038/pr.2018.9
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DOI |
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10.1038/pr.2018.9
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Short Title |
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Pediatr Res
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