EFFICACY OF ALIROCUMAB IN 1,191 PATIENTS WITH A WIDE SPECTRUM OF MUTATIONS IN GENES CAUSATIVE FOR FAMILIAL HYPERCHOLESTEROLEMIA

Authors: John J.P. Kastelein, Gisle Langslet, Paul Hopkins, Joep Defesche, Werner Seiz, Marie Baccara-Dinet, Sara Hamon, Poulabi Banerjee, Claudia Stefanutti, Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA, Sanofi, Montpellier, France Background: Next Generation Sequencing was performed to examine treatment response with alirocumab in patients carrying one or more causative mutation(s) in five familial …

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Efficacy of Alirocumab in 1191 Patients with a Wide Spectrum of Mutations in Genes Causative for Familial Hypercholesterolemia

Familial hypercholesterolemia (FH) is characterized by high levels of low-density lipoprotein cholesterol (LDL-C) and increased risk of developing premature coronary heart disease. The prevalence of heterozygous and homozygous FH has been estimated to be 1:200 to 1:500  and 1:160,000 to 1:1,000,000, respectively. In plasma, LDL-C levels are regulated via apolipoprotein …

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