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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