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" ... Brown and Goldstein’s own transformative work on cholesterol transport was similarly rooted in a small population of patients – homozygous familial hypercholesterolemia [FH]-- with an extreme phenotype -- extraordinarily high lipid levels. Their discoveries contributed to the development of the statins, medicines now taken by millions of people with elevated cholesterol to lower lipids and reduce cardiac risk. (Sadly, the patients with homozygous FH not only have the highest levels of lipids, but these lipid levels are only minimally impacted by statins; however, there’s hope these patients could derive additional lipid-lowering benefit from the new drugs targeting PCSK9.) ... "
" ... Panel members appeared to largely agree with one panel member, who explained that his yes vote was "specific for this condition" (homozygous FH) only. He said he could accept the "trade-off between a near-certain early demise versus the possibility of liver disease." ... "