Search results for "Real-world study"

showing 1 items of 1 documents

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)
researchProduct