Familial Hypercholesterolemia

LDL-cholesterol lowering and clinical outcomes in hypercholesterolemic subjects with and without a familial hypercholesterolemia phenotype: Analysis from the secondary prevention 4S trial

(2021) Antonio J. Vallejo-Vaz et al.   ATHEROSCLEROSIS

A meta-analysis of medications directed against PCSK9 in familial hypercholesterolemia

(2021) Julia Brandts et al.   ATHEROSCLEROSIS

Pathophysiology and inhibition of cholesteryl ester transfer protein for prevention of cardiovascular diseases: An update

(2021) Surojit Banerjee et al.   DRUG DISCOVERY TODAY

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

An oral antisense oligonucleotide for PCSK9 inhibition

(2021) Peter Gennemark et al.   Science Translational Medicine

LDL-Cholesterol Lowering Strategies and Population Health - Time to Move to a Cumulative Exposure Model

(2020) Julia Brandts et al.   CIRCULATION

Prevalence of Familial Hypercholesterolemia Among the General Population and Patients With Atherosclerotic Cardiovascular Disease

(2020) Pengwei Hu et al.   CIRCULATION

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

(2020) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

Angiopoietin-like protein 3, an emerging cardiometabolic therapy target with systemic and cell-autonomous functions

(2020) Hanna Ruhanen et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR AND CELL BIOLOGY OF LIPIDS

Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia

(2020) Dirk J. Blom et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Vupanorsen, an N-acetyl galactosamine-conjugated antisense drug to ANGPTL3 mRNA, lowers triglycerides and atherogenic lipoproteins in patients with diabetes, hepatic steatosis, and hypertriglyceridaemia

(2020) Daniel Gaudet et al.   EUROPEAN HEART JOURNAL

Time Course of LDL Cholesterol Exposure and Cardiovascular Disease Event Risk

(2020) Michael J. Domanski et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Evinacumab for Homozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficacy and safety of bempedoic acid in patients with heterozygous familial hypercholesterolemia: Analysis of pooled patient-level data from phase 3 clinical trials

(2020) P.B. Duell et al.   ATHEROSCLEROSIS

Safety and Efficacy of Bempedoic Acid to Reduce LDL Cholesterol

(2019) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety, Tolerability And Ldl-C Reduction With A Novel Anti-Pcsk9 Recombinant Fusion Protein (Lib003): Results Of A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study

(2019) E. Stein et al.   ATHEROSCLEROSIS

Inclisiran Durably Lowers Ldl-C And Pcsk9 Expression In Subjects With Homozygous Familial Hypercholesterolaemia: The Orion-2 Pilot Study

(2019) F. Raal et al.   ATHEROSCLEROSIS

In Vivo AAV-CRISPR/Cas9-Mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia

(2019) Huan Zhao et al.   CIRCULATION

Effect of Bempedoic Acid vs Placebo Added to Maximally Tolerated Statins on Low-Density Lipoprotein Cholesterol in Patients at High Risk for Cardiovascular Disease

(2019) Anne C. Goldberg et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

20-Year Follow-up of Statins in Children with Familial Hypercholesterolemia

(2019) Ilse K. Luirink et al.   NEW ENGLAND JOURNAL OF MEDICINE

Reduced Blood Lipid Levels With In Vivo CRISPR-Cas9 Base Editing of ANGPTL3

(2018) Alexandra C. Chadwick et al.   CIRCULATION

ANGPTL3 Deficiency and Protection Against Coronary Artery Disease

(2017) Nathan O. Stitziel et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Evacetrapib and Cardiovascular Outcomes in High-Risk Vascular Disease

(2017) A. Michael Lincoff et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides

(2017) Mark J. Graham et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effects of Anacetrapib in Patients with Atherosclerotic Vascular Disease

(2017)   NEW ENGLAND JOURNAL OF MEDICINE

Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolaemia: an interim subset analysis of the open-label TAUSSIG study

(2017) Frederick J Raal et al.   Lancet Diabetes & Endocrinology

Familial hypercholesterolemia/autosomal dominant hypercholesterolemia: Molecular defects, the LDL-C continuum, and gradients of phenotypic severity

(2016) JoAnne M. Foody et al.   Journal of Clinical Lipidology

Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys

(2016) Rosie Z. Yu et al.   Nucleic Acid Therapeutics

Prevalence and management of familial hypercholesterolaemia in coronary patients: An analysis of EUROASPIRE IV, a study of the European Society of Cardiology

(2015) Guy De Backer et al.   ATHEROSCLEROSIS

Fibronectin-based scaffold domain proteins that bind myostatin: a patent evaluation of WO2014043344

(2015) Ryan G Walker et al.   EXPERT OPINION ON THERAPEUTIC PATENTS

Cholesterol ester transfer protein inhibition by TA-8995 in patients with mild dyslipidaemia (TULIP): a randomised, double-blind, placebo-controlled phase 2 trial

(2015) G Kees Hovingh et al.   LANCET

Anacetrapib as lipid-modifying therapy in patients with heterozygous familial hypercholesterolaemia (REALIZE): a randomised, double-blind, placebo-controlled, phase 3 study

(2015) John J P Kastelein et al.   LANCET

Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial

(2015) Frederick J Raal et al.   LANCET

Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA

(2014) Hiroshi Nishimasu et al.   CELL

Lipoprotein Apheresis in the Management of Familial Hypercholesterolaemia: Historical Perspective and Recent Advances

(2014) Claudia Stefanutti et al.   Current Atherosclerosis Reports

Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society

(2014) M. Cuchel et al.   EUROPEAN HEART JOURNAL

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Acute Kidney Injury During Therapy With an Antisense Oligonucleotide Directed Against PCSK9

(2013) Eveline P. van Poelgeest et al.   AMERICAN JOURNAL OF KIDNEY DISEASES

On- and Off-Target Pharmacology of Torcetrapib

(2012) Douglas G. Johns et al.   DRUGS

Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study

(2012) Marina Cuchel et al.   LANCET

Effects of Dalcetrapib in Patients with a Recent Acute Coronary Syndrome

(2012) Gregory G. Schwartz et al.   NEW ENGLAND JOURNAL OF MEDICINE

Origins and Mechanisms of miRNAs and siRNAs

(2009) Richard W. Carthew et al.   CELL