Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia

Author:

Ajufo Ezim,Cuchel Marina

Publisher

Springer Science and Business Media LLC

Subject

Cardiology and Cardiovascular Medicine

Reference62 articles.

1. Sjouke B, Kusters DM, Kindt I, Besseling J, Defesche JC, Sijbrands EJG, et al. Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome. Eur Heart J. 2015;36:560–5.

2. Cuchel M, Bruckert E, Ginsberg HN, Raal FJ, Santos RD, Hegele RA, et al. 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. Eur Heart J England. 2014;35:2146–57.

3. Goldstein JL, Brown MS. The LDL receptor defect in familial hypercholesterolemia. Implications for pathogenesis and therapy. Med Clin North Am UNITED STATES. 1982;66:335–62.

4. Kolansky DM, Cuchel M, Clark BJ, Paridon S, McCrindle BW, Wiegers SE, et al. Longitudinal evaluation and assessment of cardiovascular disease in patients with homozygous familial hypercholesterolemia. Am J Cardiol. 2008;102:1438–43.

5. Raal FJ, Honarpour N, Blom DJ, Hovingh GK, Xu F, Scott R, et al. Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA part B): a randomised, double-blind, placebo-controlled trial. Lancet. 2014/10/06 ed. Carbohydrate and Lipid Metabolism Research Unit, Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa. Amgen Inc, One Amgen Center Drive, Thousand Oaks, CA, USA. Division of Lipidology, Department of Medicine, University of; 2015;385:341–50.

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