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Highlights Report: 2014 FH Global Summit

Highlights Report: 2014 FH Global Summit

The FH Global Summit offers a unique opportunity for all players in the healthcare arena, academia, government and patient advocacy groups, to act together to catalyse progress in FH care. Sooner not later: Children are the driver for improving FH care Top of the agenda…

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Unmet clinical needs in cholesterol lowering

Unmet clinical needs in cholesterol lowering

Recent decades has seen marked improvement in reducing rates of heart disease and stroke (cardiovascular disease), particularly in developed nations.1 High levels of low-density lipoprotein cholesterol (LDL-C), or ‘bad cholesterol’ are considered a major risk factor for cardiovascular disease. International guidelines state that reduction of…

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PCSK9 inhibition

PCSK9 inhibition

A raised level of low-density lipoprotein cholesterol (LDL-C) is one of the most important risk factors for cardiovascular disease. Despite success in recent decades in lowering LDL-C with statins and other lipid-lowering drugs, considerable risk remains for future cardiovascular events even in people who are…

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Familial hypercholesterolaemia (FH)

Familial hypercholesterolaemia (FH)

Why is cholesterol important? Cholesterol, with other fats such as triglycerides, plays a vital role in the structure and function of cells. However, too much cholesterol in the blood (hypercholesterolaemia) is a risk factor for early heart disease, heart attack and stroke. Cholesterol is transported…

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PlayProfessor Gerald Watts discusses the 10 countries project in FH

Professor Gerald Watts discusses the 10 countries project in FH

The International Atherosclerosis Society has begun a study in Asia and the Pacific Rim to provide the first comprehensive investigation of the worlds commonest genetic disorder, familial hypercholesterolaemia (FH) in the region. FH results in very high levels of low density lipoprotein cholesterol (LDL-C) and…

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PlayUnmet needs in children with FH

Unmet needs in children with FH

Identification and treatment to reduce low density lipoprotein cholesterol (LDL-C) of children with familial hypercholesterolaemia (FH) is vital to reduce the risk of cardiovascular death in early adulthood. FH is the most common genetic disorder in the world. Parents and healthcare professionals need to be…

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TESLA in brief

TESLA in brief

TESLA was a randomised, double-blind, placebo-controlled trial of the PCSK9 inhibitor, evolocumab, in 50 patients (49 received treatment) with homozygous FH (mean LDL-C at baseline 9.0 mmol/L). All were on statins and 91% also received ezetimibe. LDL receptor mutations were identified in 45 (92%) patients;…

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RUTHERFORD-2 in brief

RUTHERFORD-2 in brief

RUTHERFORD-2 was a large, randomised, placebo-controlled trial of the PCSK9 inhibitor, evolocumab, in 331 patients with heterozygous familial hypercholesterolaemia (mean LDL-C at baseline 3.9-4.2 mmol/L). All were on statins and 62% were also on ezetimibe. FH-causing mutations were identified in 80% of patients, most were…

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PCSK9 inhibitors and FH: future for standard of care?

PCSK9 inhibitors and FH: future for standard of care?

Two recent trials in FH patients – RUTHERFORD-2 and TESLA – have raised the focus of the PCSK9 inhibitors in FH.  In an accompanying Lancet editorial, Profs. Raul Santos (Sao Paulo, Brazil) and Gerald Watts (Perth, Australia) have intimidated that PCSK9 inhibitors might represent the…

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Heterogeneity in FH: What are the implications for management?

Heterogeneity in FH: What are the implications for management?

According to Professor Frederick Raal, South Africa, irrespective of genotype, what matters is reducing the LDL-C burden with lipid-lowering therapy. Familial hypercholesterolemia (FH) is an autosomal co-dominant disorder usually resulting from mutations in the LDL receptor (LDLR) gene characterised by elevated levels of LDL-cholesterol (LDL-C)…

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Alirocumab and Lp(a): Pooled analysis of Phase II trials

Alirocumab and Lp(a): Pooled analysis of Phase II trials

Alirocumab 150 mg every 2 weeks reduced Lp(a) by 30% compared with placebo, based on a pooled analysis of 3 phase II trials in patients treated with background lipid-lowering treatment.1 These findings were consistent with a pooled analysis of evolocumab phase II trials,2 suggesting a…

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Mechanistic insights from PCSK9 LOF mutations

Mechanistic insights from PCSK9 LOF mutations

Mechanistic insights from PCSK9 LOF mutations with a particular focus on data from the Copenhagen studies, were discussed by Professor Anne Tybjaerg-Hansen, Rigshospitalet, Copenhagen University Hospital, and University of Copenhagen, Denmark. Seminal studies showed that carriage of LOF mutations was associated reduction in LDL cholesterol…

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How can we optimise PCSK9-targeted therapies?

How can we optimise PCSK9-targeted therapies?

Highly relevant to these discussions was how to optimise the use of PCSK9-targeted therapies, discussed from both European (Professor Luís Masana, University Rovira and Virgili, Reus-Tarragona, Spain) and North American (Professor Henry Ginsberg, Columbia University, New York, USA) perspectives, from the guidelines perspectives. There are…

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Evidence for the effects of PCSK9 beyond the liver

Evidence for the effects of PCSK9 beyond the liver

Evidence for the effects of PCSK9 beyond the liver was discussed by Professor Bertrand Cariou, INSERM UMR1087, l’Institut du Thorax, Nantes, France. A seminal paper showed that PCSK9 exerts hypocholesterolaemic effects via action in both the liver and intestine; in the latter case, PCSK9 modulates…

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Why new NLA guidelines for dyslipidaemia? A view from the authors

Why new NLA guidelines for dyslipidaemia? A view from the authors

The US National Lipid Association (NLA) has just released recommendations for management of dyslipidaemia. Matthew K. Ito, Professor of Pharmacy Practice, Portland, Oregon and one of the lead authors, discusses thinking behind these recommendations, and what are the implications for clinicians. •                Why has the…

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Reducing the burden of Disease and Death from Familial Hypercholesterolemia: A Call to Action

Reducing the burden of Disease and Death from Familial Hypercholesterolemia: A Call to Action

Familial hypercholesterolemia (FH) is a genetic disease characterized by substantial elevations of low-density lipoprotein cholesterol, unrelated to diet or lifestyle. Untreated FH patients have 20 times the risk of developing coronary artery disease, compared to the general population. Estimates indicate that as many as 1…

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Paediatric FH: do you need a genetic diagnosis?

Paediatric FH: do you need a genetic diagnosis?

Familial hypercholesterolaemia (FH) is characterised clinically by life-long elevated levels of low-density-lipoprotein cholesterol (LDL-C) and increased risk of premature cardiovascular disease.  FH is predominantly due to mutations in the gene encoding the LDL receptor, and less commonly to mutations in the APOB or PCSK9 genes….

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PlayTake home messages from ACC2014

Take home messages from ACC2014

Results of studies presented in 2014, show that PCSK9 inhibitors: 1) very effectively reduce low density lipoprotein cholesterol (LDL-C); and 2) show consistently predictable safety profiles. The positive results of short-term studies are now being reported in longer terms and larger clinical studies, Dr Evan…

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Lowering LDL-C : at what level would this impact the benefit vs. risk ratio?

Lowering LDL-C : at what level would this impact the benefit vs. risk ratio?

Emeritus Research Professor, Institut de recherches cliniques de Montréal (IRCM), Canada Potent inhibitors of pro-protein convertase-subtilisin-kexin-9 (PCSK9) have the capacity to reduce plasma low-density lipoprotein cholesterol (LDL-C) to unprecedentedly low levels, especially if combined with statins. This discovery raises several important questions; how low should…

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