Research graph
References from Current, novel and emerging lipid-lowering therapies in pediatric familial hypercholesterolemia. Local targets link to admitted publications; unresolved targets remain external evidence.
Familial defective apolipoprotein B-100: low density lipoproteins with abnormal receptor binding
10.1073/pnas.84.19.6919 · 1987 · External reference
Mutations and polymorphisms in the proprotein convertase subtilisin kexin 9 (PCSK9) gene in cholesterol metabolism and disease
10.1002/humu.20882 · 2009 · External reference
Prevalence of familial hypercholesterolemia among the general population and patients with atherosclerotic cardiovascular disease: a systematic review and meta-analysis
10.1161/circulationaha.119.044795 · 2020 · External reference
Low-density lipoproteins cause atherosclerotic cardiovascular disease. 1. Evidence from genetic, epidemiologic, and clinical studies. A consensus statement from the European Atherosclerosis Society Consensus Panel
10.1093/eurheartj/ehx144 · 2017 · External reference
20-year follow-up of statins in children with familial hypercholesterolemia
10.1056/nejmoa1816454 · 2019 · External reference
Statin treatment in children with familial hypercholesterolemia: the younger, the better
10.1161/circulationaha.106.671016 · 2007 · External reference
Familial hypercholesterolaemia in children and adolescents: gaining decades of life by optimizing detection and treatment
10.1093/eurheartj/ehv157 · 2015 · External reference
Cholesterol-lowering therapy from childhood/adolescence and long-term outcomes in familial hypercholesterolaemia: the SAFEHEART study
10.1093/eurheartj/ehag349 · 2026 · External reference
Familial hypercholesterolaemia in children and adolescents: a European Atherosclerosis Society consensus statement
10.1093/eurheartj/ehag382 · 2026 · External reference
2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance
10.1093/eurheartj/ehad197 · 2023 · External reference
Management of children with heterozygous familial hypercholesterolaemia worldwide: a meta-analysis
10.1093/ehjopen/oeaf001 · 2025 · External reference
Treatment of pediatric heterozygous familial hypercholesterolemia 7 years after the EAS recommendations: real-world results from a large French cohort
10.1016/j.arcped.2024.01.004 · 2024 · External reference
The UK Paediatric Familial Hypercholesterolaemia Register: preliminary data
10.1136/archdischild-2015-308570 · 2017 · External reference
New and emerging therapies for reduction of LDL-cholesterol and apolipoprotein B: JACC Focus Seminar 1/4
10.1016/j.jacc.2020.11.079 · 2021 · External reference
Novel therapeutic targets and agents for pediatric dyslipidemia
10.1177/20420188211058323 · 2021 · External reference
Statin-associated symptoms and statin intolerance in children with familial hypercholesterolemia: insights from 15 years of clinical practice
10.1016/j.atherosclerosis.2026.120659 · 2026 · External reference
Efficacy and safety of ezetimibe monotherapy in children with heterozygous familial or nonfamilial hypercholesterolemia
10.1016/j.jpeds.2015.02.043 · 2015 · External reference
Ezetimibe: integrating established use with new evidence - a comprehensive review
10.1007/s11883-024-01248-w · 2024 · External reference
Practice of lipoprotein apheresis and short-term efficacy in children with homozygous familial hypercholesterolemia: data from an international registry
10.1016/j.atherosclerosis.2020.01.031 · 2020 · External reference
LDL-cholesterol reduction in patients with hypercholesterolemia by modulation of adenosine triphosphate-citrate lyase and adenosine monophosphate-activated protein kinase
10.1097/mol.0000000000000091 · 2014 · External reference
Liver-specific ATP-citrate lyase inhibition by bempedoic acid decreases LDL-C and attenuates atherosclerosis
10.1038/ncomms13457 · 2016 · External reference
Bempedoic acid plus ezetimibe fixed-dose combination in patients with hypercholesterolemia and high CVD risk treated with maximally tolerated statin therapy
10.1177/2047487319864671 · 2020 · External reference
Efficacy and safety of bempedoic acid in patients with hypercholesterolemia and statin intolerance
10.1161/jaha.118.011662 · 2019 · External reference
Effect of bempedoic acid vs placebo added to maximally tolerated statins on low-density lipoprotein cholesterol in patients at high risk for cardiovascular disease: the CLEAR Wisdom Randomized Clinical Trial
10.1001/jama.2019.16585 · 2019 · External reference
Oligonucleotide therapeutics - a new class of cholesterol-lowering drugs
10.1056/nejmp1614154 · 2017 · External reference
Alirocumab in pediatric patients with heterozygous familial hypercholesterolemia: a randomized clinical trial
10.1001/jamapediatrics.2023.6477 · 2024 · External reference
Long-term evolocumab in patients with familial hypercholesterolemia
10.1016/j.jacc.2019.12.020 · 2020 · External reference
Efficacy and safety of inclisiran in adolescents with heterozygous familial hypercholesterolaemia (ORION-16): a two-part, randomised, multicentre clinical trial
10.1016/s2213-8587(25)00351-1 · 2026 · External reference
Efficacy and safety of inclisiran in adolescents with genetically confirmed homozygous familial hypercholesterolemia: results from the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial
10.1161/circulationaha.124.073233 · 2025 · External reference
Lerodalcibep and evolocumab for the treatment of homozygous familial hypercholesterolaemia with PCSK9 inhibition (LIBerate-HoFH): a phase 3, randomised, open-label, crossover, noninferiority trial
10.1016/s2213-8587(24)00313-9 · 2025 · External reference
Evolocumab treatment in pediatric patients with homozygous familial hypercholesterolemia: pooled data from three open-label studies
10.1161/atvbaha.123.320268 · 2024 · External reference
Efficacy and safety of oal PCSK9 inhibitor enlicitide in adults with heterozygous familial hypercholesterolemia: a randomized clinical trial
10.1001/jama.2025.20620 · 2026 · External reference
A placebo-controlled trial of the oral PCSK9 inhibitor enlicitide
10.1056/nejmoa2511002 · 2026 · External reference
ANGPTL3 blockade with a human monoclonal antibody reduces plasma lipids in dyslipidemic mice and monkeys
10.1194/jlr.m054890 · 2015 · External reference
Evinacumab for pediatric patients with homozygous familial hypercholesterolemia
10.1161/circulationaha.123.065529 · 2024 · External reference
Zodasiran, an RNAi therapeutic targeting ANGPTL3, for treating patients with homozygous familial hypercholesterolaemia (GATEWAY): an open-label, randomised, phase 2 trial
10.1016/s2213-8587(25)00290-6 · 2026 · External reference
Lomitapide for the treatment of hypercholesterolemia
10.1080/14656566.2017.1340941 · 2017 · External reference
Microsomal triglyceride transfer protein and its role in apoB-lipoprotein assembly
10.1194/jlr.r200014-jlr200 · 2003 · External reference
Lomitapide for the treatment of paediatric patients with homozygous familial hypercholesterolaemia (APH-19): results from the efficacy phase of an open-label, multicentre, phase 3 study
10.1016/s2213-8587(24)00233-x · 2024 · External reference
Real-world outcomes with lomitapide use in paediatric patients with homozygous familial hypercholesterolaemia
10.1007/s12325-019-00985-8 · 2019 · External reference
Impact of repeated dietary counseling between infancy and 14 years of age on dietary intakes and serum lipids and lipoproteins: the STRIP study
10.1161/circulationaha.107.699447 · 2007 · External reference
Long-term safety and efficacy of a cholesterol-lowering diet in children with elevated low-density lipoprotein cholesterol: seven-year results of the Dietary Intervention Study in Children (DISC)
10.1542/peds.107.2.256 · 2001 · External reference
Evinacumab and reduced lipoprotein apheresis in pediatric homozygous familial hypercholesterolemia: a retrospective study on LDL-C
10.1016/j.atherosclerosis.2025.119234 · 2025 · External reference
The therapeutic effect of liver transplantation in 14 children with homozygous familial hypercholesterolemia: a prospective cohort: liver transplant for familial hypercholesterolemia
10.1016/j.jacl.2024.08.008 · 2024 · External reference
Liver transplantation in severe homozygous familial hypercholesterolaemia: a scoping review
10.1016/j.atherosclerosis.2026.120730 · 2026 · External reference
Recent advances in gene therapy for familial hypercholesterolemia: an update review
10.3390/jcm11226773 · 2022 · External reference
Gene therapy for paediatric homozygous familial hypercholesterolaemia
10.1016/j.hlc.2023.01.017 · 2023 · External reference
Effect of bempedoic acid vs placebo added to maximally tolerated statins on low-density lipoprotein cholesterol in patients at high risk for cardiovascular disease: the CLEAR Wisdom Randomized Clinical Trial
10.1001/jama.2019.16585 · ExternalCitation · doi-reference
Efficacy and safety of oal PCSK9 inhibitor enlicitide in adults with heterozygous familial hypercholesterolemia: a randomized clinical trial
10.1001/jama.2025.20620 · ExternalCitation · doi-reference
Alirocumab in pediatric patients with heterozygous familial hypercholesterolemia: a randomized clinical trial
10.1001/jamapediatrics.2023.6477 · ExternalCitation · doi-reference
Mutations and polymorphisms in the proprotein convertase subtilisin kexin 9 (PCSK9) gene in cholesterol metabolism and disease
10.1002/humu.20882 · ExternalCitation · doi-reference
Ezetimibe: integrating established use with new evidence - a comprehensive review
10.1007/s11883-024-01248-w · ExternalCitation · doi-reference
Real-world outcomes with lomitapide use in paediatric patients with homozygous familial hypercholesterolaemia
10.1007/s12325-019-00985-8 · ExternalCitation · doi-reference
Treatment of pediatric heterozygous familial hypercholesterolemia 7 years after the EAS recommendations: real-world results from a large French cohort
10.1016/j.arcped.2024.01.004 · ExternalCitation · doi-reference
Practice of lipoprotein apheresis and short-term efficacy in children with homozygous familial hypercholesterolemia: data from an international registry
10.1016/j.atherosclerosis.2020.01.031 · ExternalCitation · doi-reference
Evinacumab and reduced lipoprotein apheresis in pediatric homozygous familial hypercholesterolemia: a retrospective study on LDL-C
10.1016/j.atherosclerosis.2025.119234 · ExternalCitation · doi-reference
Statin-associated symptoms and statin intolerance in children with familial hypercholesterolemia: insights from 15 years of clinical practice
10.1016/j.atherosclerosis.2026.120659 · ExternalCitation · doi-reference
Liver transplantation in severe homozygous familial hypercholesterolaemia: a scoping review
10.1016/j.atherosclerosis.2026.120730 · ExternalCitation · doi-reference
Gene therapy for paediatric homozygous familial hypercholesterolaemia
10.1016/j.hlc.2023.01.017 · ExternalCitation · doi-reference
Long-term evolocumab in patients with familial hypercholesterolemia
10.1016/j.jacc.2019.12.020 · ExternalCitation · doi-reference
New and emerging therapies for reduction of LDL-cholesterol and apolipoprotein B: JACC Focus Seminar 1/4
10.1016/j.jacc.2020.11.079 · ExternalCitation · doi-reference
The therapeutic effect of liver transplantation in 14 children with homozygous familial hypercholesterolemia: a prospective cohort: liver transplant for familial hypercholesterolemia
10.1016/j.jacl.2024.08.008 · ExternalCitation · doi-reference
Efficacy and safety of ezetimibe monotherapy in children with heterozygous familial or nonfamilial hypercholesterolemia
10.1016/j.jpeds.2015.02.043 · ExternalCitation · doi-reference
Lomitapide for the treatment of paediatric patients with homozygous familial hypercholesterolaemia (APH-19): results from the efficacy phase of an open-label, multicentre, phase 3 study
10.1016/s2213-8587(24)00233-x · ExternalCitation · doi-reference
Lerodalcibep and evolocumab for the treatment of homozygous familial hypercholesterolaemia with PCSK9 inhibition (LIBerate-HoFH): a phase 3, randomised, open-label, crossover, noninferiority trial
10.1016/s2213-8587(24)00313-9 · ExternalCitation · doi-reference
Zodasiran, an RNAi therapeutic targeting ANGPTL3, for treating patients with homozygous familial hypercholesterolaemia (GATEWAY): an open-label, randomised, phase 2 trial
10.1016/s2213-8587(25)00290-6 · ExternalCitation · doi-reference
Efficacy and safety of inclisiran in adolescents with heterozygous familial hypercholesterolaemia (ORION-16): a two-part, randomised, multicentre clinical trial
10.1016/s2213-8587(25)00351-1 · ExternalCitation · doi-reference
Liver-specific ATP-citrate lyase inhibition by bempedoic acid decreases LDL-C and attenuates atherosclerosis
10.1038/ncomms13457 · ExternalCitation · doi-reference
20-year follow-up of statins in children with familial hypercholesterolemia
10.1056/nejmoa1816454 · ExternalCitation · doi-reference
A placebo-controlled trial of the oral PCSK9 inhibitor enlicitide
10.1056/nejmoa2511002 · ExternalCitation · doi-reference
Oligonucleotide therapeutics - a new class of cholesterol-lowering drugs
10.1056/nejmp1614154 · ExternalCitation · doi-reference
Familial defective apolipoprotein B-100: low density lipoproteins with abnormal receptor binding
10.1073/pnas.84.19.6919 · ExternalCitation · doi-reference
Lomitapide for the treatment of hypercholesterolemia
10.1080/14656566.2017.1340941 · ExternalCitation · doi-reference
Management of children with heterozygous familial hypercholesterolaemia worldwide: a meta-analysis
10.1093/ehjopen/oeaf001 · ExternalCitation · doi-reference
2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance
10.1093/eurheartj/ehad197 · ExternalCitation · doi-reference
Cholesterol-lowering therapy from childhood/adolescence and long-term outcomes in familial hypercholesterolaemia: the SAFEHEART study
10.1093/eurheartj/ehag349 · ExternalCitation · doi-reference
Familial hypercholesterolaemia in children and adolescents: a European Atherosclerosis Society consensus statement
10.1093/eurheartj/ehag382 · ExternalCitation · doi-reference
Familial hypercholesterolaemia in children and adolescents: gaining decades of life by optimizing detection and treatment
10.1093/eurheartj/ehv157 · ExternalCitation · doi-reference
Low-density lipoproteins cause atherosclerotic cardiovascular disease. 1. Evidence from genetic, epidemiologic, and clinical studies. A consensus statement from the European Atherosclerosis Society Consensus Panel
10.1093/eurheartj/ehx144 · ExternalCitation · doi-reference
LDL-cholesterol reduction in patients with hypercholesterolemia by modulation of adenosine triphosphate-citrate lyase and adenosine monophosphate-activated protein kinase
10.1097/mol.0000000000000091 · ExternalCitation · doi-reference
The UK Paediatric Familial Hypercholesterolaemia Register: preliminary data
10.1136/archdischild-2015-308570 · ExternalCitation · doi-reference
Evolocumab treatment in pediatric patients with homozygous familial hypercholesterolemia: pooled data from three open-label studies
10.1161/atvbaha.123.320268 · ExternalCitation · doi-reference
Statin treatment in children with familial hypercholesterolemia: the younger, the better
10.1161/circulationaha.106.671016 · ExternalCitation · doi-reference
Impact of repeated dietary counseling between infancy and 14 years of age on dietary intakes and serum lipids and lipoproteins: the STRIP study
10.1161/circulationaha.107.699447 · ExternalCitation · doi-reference
Prevalence of familial hypercholesterolemia among the general population and patients with atherosclerotic cardiovascular disease: a systematic review and meta-analysis
10.1161/circulationaha.119.044795 · ExternalCitation · doi-reference
Evinacumab for pediatric patients with homozygous familial hypercholesterolemia
10.1161/circulationaha.123.065529 · ExternalCitation · doi-reference
Efficacy and safety of inclisiran in adolescents with genetically confirmed homozygous familial hypercholesterolemia: results from the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial
10.1161/circulationaha.124.073233 · ExternalCitation · doi-reference
Efficacy and safety of bempedoic acid in patients with hypercholesterolemia and statin intolerance
10.1161/jaha.118.011662 · ExternalCitation · doi-reference
Novel therapeutic targets and agents for pediatric dyslipidemia
10.1177/20420188211058323 · ExternalCitation · doi-reference
Bempedoic acid plus ezetimibe fixed-dose combination in patients with hypercholesterolemia and high CVD risk treated with maximally tolerated statin therapy
10.1177/2047487319864671 · ExternalCitation · doi-reference
ANGPTL3 blockade with a human monoclonal antibody reduces plasma lipids in dyslipidemic mice and monkeys
10.1194/jlr.m054890 · ExternalCitation · doi-reference
Microsomal triglyceride transfer protein and its role in apoB-lipoprotein assembly
10.1194/jlr.r200014-jlr200 · ExternalCitation · doi-reference
Long-term safety and efficacy of a cholesterol-lowering diet in children with elevated low-density lipoprotein cholesterol: seven-year results of the Dietary Intervention Study in Children (DISC)
10.1542/peds.107.2.256 · ExternalCitation · doi-reference
Recent advances in gene therapy for familial hypercholesterolemia: an update review
10.3390/jcm11226773 · ExternalCitation · doi-reference
