Familial hypercholesterolaemia: who works on it

One card per institution. Pick a target or a country to narrow the list.

• All targets
• PCSK9 14
• APOE 1
• PPAR 0
• ANGPTL3 3
• CD40 0
• AMPK 0
• MAPK 0
• TP53 0
• NRF2 0
• TNF 0
• BCL-2 0
• ERK 0
• mTOR 0
• NLRP3 1
• ALK 0
• EGFR 0
• GLP-1 1
• HER2 0
• IL-6 0
• JNK 0
• SIRT1 0
• STAT3 0
• STING 0
• YAP 0
• BDNF 0
• BRCA1 0
• BRCA2 0
• CDK4/6 0
• DPP-4 0
• FOXP3 0
• HMGB1 0
• IL-17 0
• JAK 0
• KRAS 0
• LAG-3 0
• MDM2 0
• MEK 0
• PD-L1 0
• PSMA 0
• ROS1 0
• TLR4 0
• VEGF 0

20 of 20+ institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• European Commission (Horizon Europe) · HORIZON-RIA

  NEW STRATEGIES FOR THE EARLY DIAGNOSIS, RISK STRATIFICATION AND CO-MANAGEMENT OF FAMILIAL HYPERCHOLESTEROLEMIA (FH-EARLY)

  Associação para Investigação e Desenvolvimento da Faculdade de Medicina· Lisbon, Portugal· EU contribution €7,286,252· until Dec 2028· 9 countries

1 of 1 running projects, newest first. Listed amounts together: €7.3m.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Withdrawn

  Immunomodulatory Effects of PCSK9 Inhibition

  NCT05720156· Massachusetts General Hospital· Updated 26 Sept 2025

  “Lack of Feasibility”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)

  NCT07058077· Merck Sharp & Dohme LLC· Source

• A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.

  NCT05739383· Novartis Pharmaceuticals· Source

• A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-013) CORALreef Lipids

  NCT05952856· Merck Sharp & Dohme LLC· Source

• A Trial Assessing the Effectiveness of Evolocumab in Reducing the Plaque Burden in Participants With Subclinical Atheroma

  NCT07803679· Amgen· Source

• A Study to Evaluate the Pharmacodynamics of a Combination of QCZ484 and Inclisiran in Participants With Hypertension and Concomitant Hypercholesterolemia

  NCT07610278· Novartis Pharmaceuticals· Source

• A Study to Investigate LDL-cholesterol Lowering With Inclisiran Compared to Bempedoic Acid in Patients With Atherosclerotic Cardiovascular Disease.

  NCT06431763· Novartis Pharmaceuticals· Source

• A Clinical Study to Evaluate the Safety and Effectiveness of Inclisiran Sodium in Indian Patients With Primary Hypercholesterolemia or Mixed Dyslipidemia

  NCT06386419· Novartis Pharmaceuticals· Source

• Dual PCSK9 Inhibition With Inclisiran and Alirocumab in Secondary Prevention

  NCT07581808· University Medical Centre Ljubljana· Source

• Study of Efficacy, Safety, Tolerability and Quality of Life of Inclisiran (KJX839) vs Placebo, on Top of Ongoing Individually Optimized Lipid-lowering Therapy, in Participants With Hypercholesterolemia

  NCT05192941· Novartis Pharmaceuticals· Source

• A Study to Evaluate the Efficacy and Safety of Enlicitide (MK-0616, Oral PCSK9 Inhibitor) Compared With Ezetimibe or Bempedoic Acid or Ezetimibe and Bempedoic Acid in Adults With Hypercholesterolemia (MK-0616-018/CORALreef AddOn)

  NCT06450366· Merck Sharp & Dohme LLC· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Mendelian Randomization Analysis 6
• CRISPR-Cas Systems 7
• Gene Expression Profiling 15
• Whole Genome Sequencing 4
• Exome Sequencing 11
• Proteomics 6
• Metabolomics 5
• Mass Spectrometry 5
• Flow Cytometry 13
• Immunohistochemistry 18
• Xenograft Model Antitumor Assays 8
• Positron-Emission Tomography 3
• Magnetic Resonance Imaging 18
• Machine Learning 7
• Cohort Studies 75
• Genome-Wide Association Study 8

• Aortic Stenosis Hyalectan Remodeling Revealed by Proteomics and Glycoproteomics.

  Arterioscler Thromb Vasc Biol, Jul 2026

• Proteome Profiles of Hypercholesterolemic Patients with and Without FH Treated with a PCSK9 Inhibitor: A Comparison of Depleted and Nondepleted Samples in a Pilot Study.

  Proteomics Clin Appl, May 2026

• An In Vitro Functional Characterization of the Cholesterol-Transforming Blautia hominis Strain HA2291 Isolated from the Human Gut.

  Nutrients, Mar 2026

• Novel protein biomarkers associated with coronary artery disease in statin-treated patients with familial hypercholesterolemia.

  J Clin Lipidol, Apr 2017

• Integrative analysis of proteomic and transcriptomic data for identification of pathways related to simvastatin-induced hepatotoxicity.

  Proteomics, Apr 2013

• Quantitative proteomic analysis revealed lovastatin-induced perturbation of cellular pathways in HL-60 cells.

  J Proteome Res, Oct 2011

See which targets of this disease your abstract mentions, and go to the people working on them.