Article
Evinacumab for Homozygous Familial Hypercholesterolemia.
The New England journal of medicine - 20 Aug 2020
Raal Frederick J, Rosenson Robert S, Reeskamp Laurens F, Hovingh G Kees, Kastelein John J P, Rubba Paolo, Ali Shazia, Banerjee Poulabi, Chan Kuo-Chen, Gipe Daniel A, Khilla Nagwa, Pordy Robert, Weinreich David M, Yancopoulos George D, Zhang Yi, Gaudet Daniel
Abstract excerpt
BACKGROUND: Homozygous familial hypercholesterolemia is characterized by premature cardiovascular disease caused by markedly elevated levels of low-density lipoprotein (LDL) cholesterol. This disorder is associated with genetic variants that result in virtually absent (null-null) or impaired (non-null) LDL-receptor activity. Loss-of-function variants in the gene encoding angiopoietin-like 3 (ANGPTL3) are...
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