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To read the full abstract: N Engl J Med. 2020;382(16):152030. doi: 10.1056/NEJMoa1913805Short summary: Inclisiran (a small interfering double-stranded RNA against PCSK9) is superior to placebo in reducing LDL-C among individuals with heterozygous Familial Hypercholesterolemia who are already on statins and ezetimibe.Comment: Proprotein convertas...

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ey0021.12-15 | Lipid Metabolism | ESPEYB21

12.15. Cardiovascular outcomes in patients with homozygous familial hypercholesterolaemia on lipoprotein apheresis initiated during childhood: long-term follow-up of an international cohort from two registries

MD Reijman , TR Tromp , BA Hutten , GK Hovingh , DJ Blom , AL Catapano , M Cuchel , EJ Dann , A Gallo , LC Hudgins , FJ Raal , KK Ray , F Sadiq , H Soran , JW Groothoff , A Wiegman , DM Kusters

Brief Summary: Data from two large registries of children with homozygous familial hypercholesterolemia (HoFH) revealed that initiating lipoprotein apheresis in childhood, compared to pharmacotherapy, improves plasma LDL-C level and reduces cardiovascular death.Comment: Homozygous familial hypercholesterolaemia (HoFH) is a rare inherited disorder, which results in extremely elevated low-density lipoprotein cholesterol (LDL-C) levels and premature atheros...

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ey0020.10-11 | Dyslipidemia | ESPEYB20

10.11. 2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance

M Cuchel , FJ Raal , RA Hegele , K Al-Rasadi , M Arca , M Averna , E Bruckert , T Freiberger , D Gaudet , M Harada-Shiba , LC Hudgins , M Kayikcioglu , L Masana , KG Parhofer , JE Roeters van Lennep , RD Santos , ESG Stroes , GF Watts , A Wiegman , JK Stock , LS Tokgozoğlu , AL Catapano , KK Ray

Brief summary: In May 2023, the European Atherosclerosis Society (EAS) released an update to its 2014 consensus on homozygous familial hypercholesterolemia (HoFH). The 2023 statement provides updated diagnostic criteria, screening recommendations, treatment algorithms, guidance about family planning, and new insights into the genetics of the disease.Comment: Here is a brief summary of the new findings and recommendations of the updated consensus:<p c...

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ESPE Yearbook of Paediatric Endocrinology

12.15. Inclisiran for the treatment of heterozygous familial hypercholesterolemia

12.15. Cardiovascular outcomes in patients with homozygous familial hypercholesterolaemia on lipoprotein apheresis initiated during childhood: long-term follow-up of an international cohort from two registries

10.11. 2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance

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