Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study
- Author
- CUCHEL, Marina1 ; MEAGHER, Emma A1 ; STEFANUTTI, Claudia11 ; VIGNA, Giovanni B12 ; DU PLESSIS, Anna M. E13 ; PROPERT, Kathleen J1 ; SASIELA, William J14 ; BLOEDON, Leanne T1 14 ; RADER, Daniel J1 ; DU TOIT THERON, Hendrik2 ; BLOM, Dirk J3 5 ; MARAIS, A. David4 5 ; HEGELE, Robert A6 ; AVERNA, Maurizio R7 ; SIRTORI, Cesare R8 ; SHAH, Prediman K9 ; GAUDET, Daniel10
[1] Institute forTranslational Medicine and Therapeutics, Cardiovascular Institute, and Department of Medicine, University of Pennsylvania, Philadelphia, PA, United States
[2] Netcare Private Hospital, Bloemfontein, South Africa
[3] Department of Medicine, University of Cape Town, Cape Town, South Africa
[4] Department of Chemical Pathology, University of Cape Town, Cape Town, South Africa
[5] Medical Research Council of South Africa, Cape Heart Group, Cape Town, South Africa
[6] Robarts Research Institute and Schulich School of Medicine and Dentistry, University of Western Ontario, London, ON, Canada
[7] Università di Palermo, Palermo, Italy
[8] Ospedale Niguarda, Milano, Italy
[9] Division of Cardiology and Atherosclerosis Research Center, Cedars-Sinai Heart Institute, Los Angeles, CA, United States
[10] Department of Medicine, Universite de Montreal, Chicoutimi, Quebec, Canada
[11] Extracorporeal Therapeutic Techniques Unit, Immunohematology and Transfusion Medicine, Department of Molecular Medicine, University of Rome, Sapienza, Italy
[12] Department of Clinical and Experimental Medicine, Università of Ferrara, Italy
[13] Clinical Research Unit, University of Pretoria, Pretoria, South Africa
[14] Aegerion Pharmaceuticals, Cambridge, MA, United States
Phase 3 HoFH Lomitapide Study investigators - Source
Lancet (British edition). 2013, Vol 381, Num 9860, pp 40-46, 7 p ; ref : 24 ref
- CODEN
- LANCAO
- ISSN
- 0140-6736
- Scientific domain
- General medicine general surgery
- Publisher
- Elsevier, Kidlington
- Publication country
- United Kingdom
- Document type
- Article
- Language
- English
- Keyword (fr)
- Bras Cholestérol Efficacité traitement Essai clinique phase III Essai clinique Homme Homozygotie Hypercholestérolémie Inhibiteur protéique Lipoprotéine Malade Maladie héréditaire Microsome Médecine Médicament Sécurité Toxicité Transfert Triglycéride Essai ouvert Essai thérapeutique Dyslipémie Hyperlipoprotéinémie Lipide Maladie métabolique
- Keyword (en)
- Arm Cholesterol Treatment efficiency Phase III trial Clinical trial Human Homozygosity Hypercholesterolemia Protein inhibitor Lipoprotein Patient Genetic disease Microsome Medicine Drug Safety Toxicity Transfer Triglyceride Dyslipemia Hyperlipoproteinemia Lipids Metabolic diseases
- Keyword (es)
- Brazo Colesterol Eficacia tratamiento Ensayo clínico fase III Ensayo clínico Hombre Homocigosis Hipercolesterolemia Inhibidor proteico Lipoproteina Enfermo Enfermedad hereditaria Microsoma Medicina Medicamento Seguridad Toxicidad Transferencia Triglicérido Dislipemia Hiperlipoproteinemia Lípido Metabolismo patología
- Classification
- Pascal
- 002 Biological and medical sciences / 002B Medical sciences / 002B22 Metabolic diseases / 002B22A Disorders of blood lipids. Hyperlipoproteinemia
- Discipline
- Generalities in medical sciences Metabolic diseases
- Origin
- Inist-CNRS
- Database
- PASCAL
- INIST identifier
- 26780318
Sauf mention contraire ci-dessus, le contenu de cette notice bibliographique peut être utilisé dans le cadre d’une licence CC BY 4.0 Inist-CNRS / Unless otherwise stated above, the content of this bibliographic record may be used under a CC BY 4.0 licence by Inist-CNRS / A menos que se haya señalado antes, el contenido de este registro bibliográfico puede ser utilizado al amparo de una licencia CC BY 4.0 Inist-CNRS
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DOI
10.1016/S0140-6736(12)61731-0 
https://dx.doi.org/10.1016/S0140-6736(12)61731-0
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