Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study
- Authors: Cuchel, M; Meagher, E; du Toit Theron, H; Blom, D; Marais, A; Hegele, R; Cefalu, AB; Averna, M; Sirtori, C; Shah, P; Gaudet, D; Stefanutti, C; Vigna, G; Du Plessis, A; Propert, K; Sasiela, W; Bloedon, L; Rader, D; Noto, D
- Publication year: 2013
- Type: Articolo in rivista (Articolo in rivista)
- OA Link: http://hdl.handle.net/10447/74492
Abstract
Patients with homozygous familial hypercholesterolaemia respond inadequately to existing drugs. We aimed to assess the efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in adults with this disease.