{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["12(2)"],"submitter":["Ho VQT"],"pubmed_abstract":["Familial hypercholesterolaemia (FH) is a hereditary disorder characterised by elevated low-density lipoprotein cholesterol (LDL-C) levels, substantially increasing the risk of atherosclerotic cardiovascular disease. Proprotein convertase subtilisin/kexin type 9 (PCSK9) targeting therapies, including monoclonal antibodies and small interfering RNA (siRNA) agents, have emerged as effective lipid lowering therapies. To assess the efficacy and safety of PCSK9-targeting therapy on lipid biomarkers and adverse events in patients with FH, compared with placebo on the background of standard lipid-lowering therapy. A systematic review and meta-analysis were conducted, incorporating data from 23 randomised controlled trials involving adult and paediatric FH patients treated with PCSK9 inhibitors (PC"],"journal":["Open heart"],"pagination":["e003490"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12374655"],"repository":["biostudies-literature"],"pubmed_title":["PCSK9 targeting therapies for familial hypercholesterolaemia: a meta-analysis of efficacy on lipid biomarkers and safety in adults and children across 23 RCTs."],"pmcid":["PMC12374655"],"pubmed_authors":["Leucker TM","Barbagelata A","Giorgi J","Tran NB","Arrighini GS","Dandamudi M","Walia H","Downes D","Ho VQT","Nguyen N","Ferrara VZ","Ho THQ"],"additional_accession":[]},"is_claimable":false,"name":"PCSK9 targeting therapies for familial hypercholesterolaemia: a meta-analysis of efficacy on lipid biomarkers and safety in adults and children across 23 RCTs.","description":"Familial hypercholesterolaemia (FH) is a hereditary disorder characterised by elevated low-density lipoprotein cholesterol (LDL-C) levels, substantially increasing the risk of atherosclerotic cardiovascular disease. Proprotein convertase subtilisin/kexin type 9 (PCSK9) targeting therapies, including monoclonal antibodies and small interfering RNA (siRNA) agents, have emerged as effective lipid lowering therapies. To assess the efficacy and safety of PCSK9-targeting therapy on lipid biomarkers and adverse events in patients with FH, compared with placebo on the background of standard lipid-lowering therapy. A systematic review and meta-analysis were conducted, incorporating data from 23 randomised controlled trials involving adult and paediatric FH patients treated with PCSK9 inhibitors (PC","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Aug","modification":"2026-05-09T10:33:44.95Z","creation":"2026-04-08T00:46:49.044Z"},"accession":"S-EPMC12374655","cross_references":{"pubmed":["40841123"],"doi":["10.1136/openhrt-2025-003490"]}}