Gene therapy, a promising treatment for familial hypercholesterolaemia

Rotondo, Dino; Davidson, Jillian

doi:10.1097/mol.0000000000000402

Cited by 2 publications

(1 citation statement)

References 15 publications

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“…36 Liver-directed gene therapy for hoFH is currently being trialled. 37 Although LA is considered the standard care for patients with hoFH, its limited availability, high cost, duration of the procedure, and maintenance of vascular access are significant drawbacks. 38 LA also requires commitment from the patient.…”

Section: Discussionmentioning

confidence: 99%

A cross-national investigation of cardiovascular survival in homozygous familial hypercholesterolemia: The Sino-Roman Study

Stefanutti

Pang

Giacomo

et al. 2019

Journal of Clinical Lipidology

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BACKGROUND: Homozygous familial hypercholesterolemia (hoFH) is a rare inherited disorder characterized by extreme elevation of low-density lipoprotein (LDL) cholesterol, accelerated coronary artery disease, and premature death. Aggressive LDL-lowering therapies are important for survival, but these are not available worldwide. OBJECTIVE: The aim of the study was to compare and contrast cardiovascular outcomes and mortality of hoFH patients in 2 countries with disparate use of lipoprotein apheresis (LA) and modern therapies for lowering LDL cholesterol. METHODS: A retrospective study was undertaken comparing cardiovascular disease (CVD)-free survival and mortality in 44 hoFH patients who were treated with statins but not LA, from a center in Beijing, China, and 18 hoFH patients who were treated with LA and novel therapies from an early age, from a center in Rome, Italy. RESULTS: CVD-free survival and survival were significantly reduced in Chinese patients compared with the Italian patients after 30 years of follow-up (log-rank P , .01). In a pooled analysis,

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Section: Discussionmentioning

confidence: 99%