Search results for " Lomitapide"

showing 2 items of 12 documents

Long-term efficacy of lipoprotein apheresis and lomitapide in the treatment of homozygous familial hypercholesterolemia (HoFH): a cross-national retr…

2021

Abstract Background Homozygous familial hypercholesterolemia (HoFH) is a rare life-threatening condition that represents a therapeutic challenge. The vast majority of HoFH patients fail to achieve LDL-C targets when treated with the standard protocol, which associates maximally tolerated dose of lipid-lowering medications with lipoprotein apheresis (LA). Lomitapide is an emerging therapy in HoFH, but its place in the treatment algorithm is disputed because a comparison of its long-term efficacy versus LA in reducing LDL-C burden is not available. We assessed changes in long-term LDL-C burden and goals achievement in two independent HoFH patients’ cohorts, one treated with lomitapide in Ita…

medicine.medical_specialtySettore MED/09 - Medicina Interna[SDV]Life Sciences [q-bio]LipoproteinsGenetic diseaseTherapeuticsFamilial hypercholesterolemiaDiseaseLipoprotein apheresiLDLHyperlipoproteinemia Type IIchemistry.chemical_compoundLipoprotein apheresisRetrospective surveyInternal medicineCholesterol burden; Genetic disease; Homozygous hypercholesterolemia; LDL; Lipoprotein apheresis; Lomitapide; Therapeutics; Benzimidazoles; Homozygote; Humans; Lipoproteins; Retrospective Studies; Anticholesteremic Agents; Blood Component Removal; Hyperlipoproteinemia Type IImedicineHumansPharmacology (medical)Genetics (clinical)Retrospective Studiesmedicine.diagnostic_testbusiness.industryResearchAnticholesteremic AgentsHomozygous hypercholesterolemiaHomozygoteRGeneral Medicinemedicine.diseaseLomitapideLomitapidecholesterol burden; genetic disease; homozygous hypercholesterolemia; LDL; lipoprotein apheresis; lomitapide; therapeuticsCholesterol burdenchemistryCohortBlood Component RemovalMedicineTherapeutics.BenzimidazolesLipid profilebusinessLipoprotein apheresisCross nationalOrphanet Journal of Rare Diseases
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Efficacy of Long-Term Treatment of Autosomal Recessive Hypercholesterolemia With Lomitapide: A Subanalysis of the Pan-European Lomitapide Study

2022

Backgroundand aim: Autosomal recessive hypercholesterolemia (ARH) is a rare autosomal recessive disorder of low-density lipoprotein (LDL) metabolism caused by pathogenic variants in the LDLRAP1 gene. Like homozygous familial hypercholesterolemia, ARH is resistant to conventional LDL-lowering medications and causes a high risk of atherosclerotic cardiovascular diseases (ASCVDs) and aortic valve stenosis. Lomitapide is emerging as an efficacious therapy in classical HoFH, but few data are available for ARH.Results: This is a subanalysis carried out on nine ARH patients included in the Pan-European Lomitapide Study. The age at starting lomitapide was 46 (interquartile range (IQR), 39.0–65.5) y…

safetylomitapidelong-termsafety.Settore MED/09 - Medicina Internaefficacyrare diseaseReal-world studySDG 3 - Good Health and Well-beingSettore BIO/14 - FarmacologiaGeneticsMolecular MedicineLDL-C; Real-world study; autosomal recessive hypercholesterolaemia; efficacy; lomitapide; long-term; rare disease; safetyautosomal recessive hypercholesterolaemiaLDL-CGenetics (clinical)
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