Familial chylomicronaemia syndrome (FCS) is a rare autosomal recessive disorder, resulting in elevated triglycerides (TGs), abdominal pain and pancreatitis. Treatment options are limited. Lomitapide, a microsomal triglyceride transfer protein inhibitor, is approved for the treatment of homozygous familial hypercholesterolaemia. Whether its therapeutic use may be extended to FCS remains unknown. The aim of this study was to evaluate the efficacy and safety of lomitapide in adult patients with FCS.
Cefalu', A.B., D'Erasmo, L., Iannuzzo, G., Noto, D., Giammanco, A., Montali, A., et al. (2022). Efficacy and safety of lomitapide in familial chylomicronaemia syndrome. ATHEROSCLEROSIS, 359, 13-19 [10.1016/j.atherosclerosis.2022.08.017].
Efficacy and safety of lomitapide in familial chylomicronaemia syndrome
Cefalu', Angelo BaldassarePrimo
Writing – Original Draft Preparation
;Noto, Davide;Giammanco, Antonina;Barbagallo, Carlo M;Ganci, Antonina;Nardi, Emilio;Vernuccio, Federica;Caldarella, Rosalia;Ciaccio, Marcello;Averna, Maurizio
Ultimo
2022-10-01
Abstract
Familial chylomicronaemia syndrome (FCS) is a rare autosomal recessive disorder, resulting in elevated triglycerides (TGs), abdominal pain and pancreatitis. Treatment options are limited. Lomitapide, a microsomal triglyceride transfer protein inhibitor, is approved for the treatment of homozygous familial hypercholesterolaemia. Whether its therapeutic use may be extended to FCS remains unknown. The aim of this study was to evaluate the efficacy and safety of lomitapide in adult patients with FCS.
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Efficacy and safety of lomitapide in familial chylomicronaemia syndrome.pdf
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