showing 3 related works from this author
Cause of death in patients with attenuated acid sphingomyelinase deficiency: Comprehensive literature review and report of new cases
2016
1Niemann-Pick Disease Group (UK), Tyne and Wear, UK; 2Villa Metabolica, ZKJM, Medical Center, University of Mainz, Mainz, Germany; 3University of Leuven, Leuven, Belgium; 4Hospital Dr. Exequiel Gonzalez Cortes, Santiago, Chile; 5King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia; 6Academic Medical Center Hospital of Udine, Udine, Italy; 7Medical Genetics Service, HCPA/UFRGS, Porto Alegre, Brazil; 8University of California San Francisco, San Francisco, CA, USA; 9University of Paris-Diderot, Reference Center for Inborn Errors of Metabolism, Robert-Debre Hospital, Paris, France; 10Akita University School of Medicine, Akita, Japan; 11La Rabta Hospital, Tunis, Tunisia; 12San…
Elafibranor, an Agonist of the Peroxisome Proliferator-Activated Receptor-alpha and -delta, Induces Resolution of Nonalcoholic Steatohepatitis Withou…
2016
International audience; BACKGROUND & AIMS: Elafibranor is an agonist of the peroxisome proliferator-activated receptor-α and peroxisome proliferator-activated receptor-δ. Elafibranor improves insulin sensitivity, glucose homeostasis, and lipid metabolism and reduces inflammation. We assessed the safety and efficacy of elafibranor in an international, randomized, double-blind placebo-controlled trial of patients with nonalcoholic steatohepatitis (NASH).METHODS: Patients with NASH without cirrhosis were randomly assigned to groups given elafibranor 80 mg (n = 93), elafibranor 120 mg (n = 91), or placebo (n = 92) each day for 52 weeks at sites in Europe and the United States. Clinical and …
Worldwide experience of homozygous familial hypercholesterolaemia:retrospective cohort study
2022
[Background]: Homozygous familial hypercholesterolaemia (HoFH) is a rare inherited disorder resulting in extremely elevated low-density lipoprotein cholesterol levels and premature atherosclerotic cardiovascular disease (ASCVD). Current guidance about its management and prognosis stems from small studies, mostly from high-income countries. The objective of this study was to assess the clinical and genetic characteristics, as well as the impact, of current practice on health outcomes of HoFH patients globally.