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.
Long-term efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in patients with homozygous familial hypercholesterolemia / Blom, D. J.; Averna, M. R.; Meagher, E. A.; Toit Theron, H. D.; Sirtori, C. R.; Hegele, R. A.; Shah, P. K.; Gaudet, D.; Stefanutti, C.; Vigna, G. B.; Larrey, D.; Bloedon, L. T.; Foulds, P.; Rader, D. J.; Cuchel, M.. - In: CIRCULATION. - ISSN 0009-7322. - 136:3(2017), pp. 332-335. [10.1161/CIRCULATIONAHA.117.028208]
Long-term efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in patients with homozygous familial hypercholesterolemia
Stefanutti C.;
2017
Abstract
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.| File | Dimensione | Formato | |
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