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Therapeutic options for homozygous familial hypercholesterolemia: the role of Lomitapide

  • Autori: Giammanco, Antonina; Cefalù, Angelo B; Noto, Davide; Averna, Maurizio
  • Anno di pubblicazione: 2020
  • Tipologia: Articolo in rivista
  • Parole Chiave: HoFH – Lomitapide – LOWER Registry – MTP inhibition – MTP SNPs
  • OA Link:


Lomitapide (Juxtapid® in US and Lojuxta® in Europe) is the first developed inhibitor of the microsomal triglyceride transfer protein (MTP) approved as a novel drug for the management of homozygous familial hypercholesterolemia (HoFH). It acts by binding directly and selectively to MTP thus decreasing the assembly and secretion of the apo-B containing lipoproteins both in the liver and in the intestine.