0000000000540072

AUTHOR

Pamela Foulds

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Long-Term Efficacy and Safety of the Microsomal Triglyceride Transfer Protein Inhibitor Lomitapide in Patients With Homozygous Familial Hypercholeste…

2017

Homozygous familial hypercholesterolemia is a genetic disorder characterized by low-density lipoprotein (LDL)-receptor dysfunction, markedly elevated levels of LDL-cholesterol (LDL-C) and premature atherosclerosis. Patients are often poorly responsive to conventional lipid-lowering therapies that upregulate LDL-receptor expression.1 Lomitapide inhibits microsomal triglyceride transfer protein, which lipidates nascent apolipoprotein (apo)B-containing lipoproteins. In a pivotal 78-week open-label trial, lomitapide, titrated to the maximal tolerable dose, decreased LDL-C by 50% at the end of the efficacy phase (week 26) in patients with homozygous familial hypercholesterolemia.2 The principal …

AdultMalemedicine.medical_specialtySettore MED/09 - Medicina InternaApolipoprotein BSocio-culturaleFamilial hypercholesterolemia030204 cardiovascular system & hematologyGastroenterologyMicrosomal triglyceride transfer proteinLDLTimeSudden cardiac deathHyperlipoproteinemia Type II03 medical and health scienceschemistry.chemical_compound0302 clinical medicinePhysiology (medical)Internal medicinemedicineHumans030212 general & internal medicineAdverse effectlomitapidebiologybusiness.industryCholesterolAnticholesteremic AgentsCholesterol LDLlomitapide; Adult; Anticholesteremic Agents; Benzimidazoles; Carrier Proteins; Cholesterol LDL; Female; Humans; Hyperlipoproteinemia Type II; Male; Timemedicine.diseaseLomitapideCholesterolEndocrinologychemistrybiology.proteinBenzimidazolesFemalelipids (amino acids peptides and proteins)lomitapide; Cardiology and Cardiovascular Medicine; Physiology (medical)Carrier ProteinsCardiology and Cardiovascular MedicinebusinessLipoproteinCirculation
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