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Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia : a single-arm, open-label, phase 3 study

Cuchel, Marina; Meagher, Emma A.; Theron, Hendrik Du Toit; Blom, Dirk J.; Marais, A. David; Hegele, Robert A.; Averna, Maurizio R.; Sirtori, Cesare R.; Shah, Prediman K.; Gaudet, Daniel; Stefanutti, Claudia; Vigna, Giovanni B.; Du Plessis, Anna M.E.; Propert, Kathleen J.; Sasiela, William J.; Bloeden, LeAnne T.; Rader, Daniel J. (Elsevier, 2013)

BACKGROUND Patients with homozygous familial hypercholesterolaemia respond inadequately to existing drugs. We aimed to assess the effi cacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide ...

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