John R. Burnett

Injectable, oral, gene-based, and vaccine-driven PCSK9-targeted therapies: emerging clinical insights shaping the future of hypercholesterolemia management

T. M. Parameswaran, Amanda J. Hooper, John R. Burnett

1. The role of proprotein convertase subtilisin/kexin type 9 (PCSK9) in cholesterol metabolism was established over two decades ago with the discovery that rare “gain-of-function” PCSK9 missense va...

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Assessing the clinical progress of muvalaplin for reducing lipoprotein(a)

Amanda J. Hooper, P. Mihika S. Fernando, John R. Burnett

INTRODUCTION: Lipoprotein(a) [Lp(a)] is an LDL-like particle, which is synthesized and assembled in the liver, and whose plasma levels are strongly associated with, and considered to be causative of, atherosclerotic cardiovascular disease (ASCVD). Several promising pharmacological therapies that directly target Lp(a) are …

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Assessing the clinical development of zerlasiran, a small-interfering RNA for elevated lipoprotein(a)

Xuan Tang, Amanda J. Hooper, John R. Burnett

INTRODUCTION: Lipoprotein(a) [Lp(a)] is an independent, inherited risk factor for atherosclerotic cardiovascular disease (ASCVD) and aortic stenosis. Lp(a) is an LDL-like particle containing apoB-100 and apo(a). Lifestyle changes and statin therapy lower LDL-cholesterol and apoB, but do not reduce Lp(a), whereas PCSK9 …

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Emerging ANGPTL3-directed therapies for hyperlipidemia: insights from two recent phase II trials

Amanda J. Hooper, T. M. Parameswaran, John R. Burnett

1. Interest in angiopoietin-like 3 (ANGPTL3) as a potential therapeutic target for lipid-lowering intensified with the discovery 15 years ago of a kindred with the rare recessive disorder familial ...

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Incidental Finding of Severe Hypertriglyceridaemia and Diagnosis of Familial Chylomicronaemia Syndrome in a Family

Vijayakumary Thadchanamoorthy, Kavinda Dayasiri, Hemika Yoganathan, S. I. Majitha et autres

The authors declare no conflicts of interest. The data that support the findings of this study are available from the corresponding author upon reasonable request.

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Lomitapide for the treatment of pediatric homozygous familial hypercholesterolemia

Amanda J. Hooper, Damon A. Bell, John R. Burnett

INTRODUCTION: Homozygous familial hypercholesterolemia (HoFH) is a rare, inherited disorder characterized by severe LDL-hypercholesterolemia and accelerated atherosclerotic cardiovascular disease. It typically presents in childhood or adolescence, and if untreated, may be fatal in the first decades of life. The microsomal triglyceride transfer …

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An evaluation of recaticimab for the treatment of hypercholesterolemia

Xuan Tang, Amanda J. Hooper, John R. Burnett

INTRODUCTION: Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, by preventing the degradation of LDL receptors, either through interference in the binding of PCSK9 to LDL receptors or through silencing of PCSK9 at a molecular level, have revolutionized lipid-lowering treatment and offer the …

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Sepiapterin for the treatment of phenylketonuria

Robin Williams, Damon A. Bell, Amanda J. Hooper, John R. Burnett

Introduction Phenylketonuria (PKU) is an autosomal recessive inborn error of phenylalanine (Phe) metabolism resulting from deficiency of phenylalanine hydroxylase (PAH). Untreated, PKU may result in severe and irreversible intellectual impairment due to marked hyperphenylalaninemia (HPA). Guidelines recommend lifelong reduction in Phe levels, …

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Publishing and reviewing in the changing world of pathology journals

John R. Burnett, Wendy A. Cooper, Tristan Rutland, Diana Jones et autres

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Response to the letter to the editor: ‘Olezarsen, a liver-directed APOC3 ASO therapy for hypertriglyceridemia’

Amanda J. Hooper, Damon A. Bell, John R. Burnett

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Assessing the clinical potential of plozasiran, an APOC3 siRNA therapy for severe hypertriglyceridemia

Xuan Tang, Amanda J. Hooper, John R. Burnett

1. Mild-to-moderate hypertriglyceridemia (HTG), defined as a fasting plasma triglyceride (TG) of 150-499 mg/dL (1.7-5.6 mmol/L), is a common clinical finding on biochemical laboratory testing. Epid...

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Olezarsen, a liver-directed APOC3 ASO therapy for hypertriglyceridemia

Amanda J. Hooper, Damon A. Bell, John R. Burnett

INTRODUCTION: Apolipoprotein (apo)C-III, a key regulator of plasma triglyceride (TG) levels, is a prime candidate for the treatment of hypertriglyceridemia (HTG), prevention of acute pancreatitis, and reduction of future atherosclerotic cardiovascular disease (ASCVD) events. AREAS COVERED: antisense oligonucleotide (ASO). EXPERT OPINION: Olezarsen, …

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