Publications by authors named "J Lemale"
Currently available polyethylene glycol (PEG)-based preparations continue to represent a challenge in children. The aim of this study was to compare the efficacy and safety of a new low-volume PEG preparation with a conventional PEG-electrolyte solution (PEG-ES) in children and adolescents. This was a multicenter, randomized, observer-blind, parallel-group, phase 3 clinical trial, where patients were randomized between PMF104 (Clensia) and a conventional PEG-ES (Klean-Prep), and stratified by age stratum (2 to <6; 6 to < 12;12 to <18 years).
View Article and Find Full Text PDFBackground: Heterozygous familial hypercholesterolemia (HeFH) predisposes to premature cardiovascular diseases. Since 2015, the European Atherosclerosis Society has advocated initiation of statins at 8-10 years of age and a low-density lipoprotein cholesterol (LDL-C) target of <135 mg/dL. Longitudinal data from large databases on pharmacological management of pediatric HeFH are lacking.
View Article and Find Full Text PDFArticle Synopsis
- Sodium dependent multivitamin transporter (SMVT) deficiency is a rare genetic disorder that causes health issues due to a lack of certain vitamins, affecting around 10 known families.
- This study introduced 4 new patients from Algeria, all sharing a specific genetic mutation related to SMVT, confirming its harmful effects through RNA analysis.
- The patients exhibited similar symptoms, including optic atrophy, severe vomiting, and rapid neurological decline, suggesting a common genetic background due to a "founder effect."