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Zellweger spectrum disorder presenting with opsoclonus-myoclonus-ataxia syndrome: a case report on immunotherapy.
Acta Neurol Belg
January 2025
Department of Pediatrics, Neurology Unit, University of Health Sciences, Ankara Etlik City Hospital, Ankara, Turkey.
Introduction: Zellweger spectrum disorder (ZSD) refers to a group of autosomal recessive genetic disorders that affect multiple organ systems and are predominantly caused by pathogenic variants in PEX genes. ZSD present a wide clinical spectrum, ranging from the most severe form, Zellweger syndrome, to the mildest form, Heimler syndrome.
Case Report: A 14-month-old male patient was brought to our clinic with recent-onset ocular tremors and unsteady gait.
Safety, efficacy, and immunogenicity of a novel IgG degrading enzyme (KJ103): results from two randomised, blinded, phase 1 clinical trials.
Gene Ther
January 2025
Shanghai Bao Pharmaceuticals Co., Ltd., No. 28 Luoxin Road, Baoshan, Shanghai, China.
The approved intravenous adeno-associated virus (AAV) therapies are limited by the widespread prevalence of pre-existing anti-AAV antibodies in the general population, which are known to restrict patients' ability to receive gene therapy and limit transfection efficacy in vivo. To address this challenge, we have developed a novel recombinant human immunoglobulin G degrading enzyme KJ103, characterized by low immunogenicity and clinical value for the elimination of anti-AAV antibodies in gene transfer. Herein, we conducted two randomized, blinded, placebo-controlled, single ascending dose Phase I studies in China and New Zealand, to evaluate the pharmacokinetics, pharmacodynamics, safety and immunogenicity of KJ103 in healthy volunteers.
View Article and Find Full Text PDFThe association between metabolomic profiles of lifestyle and the latent phase of incident chronic kidney disease in the UK Population.
Sci Rep
January 2025
NHC Key Lab of Hormones and Development and Tianjin Key Lab of Metabolic Diseases, Tianjin Medical University Chu Hsien-I Memorial Hospital & Institute of Endocrinology, Tianjin, 300134, China.
Chronic kidney disease (CKD) is a global health challenge associated with lifestyle factors such as diet, alcohol, BMI, smoking, sleep, and physical activity. Metabolomics, especially nuclear magnetic resonance(NMR), offers insights into metabolic profiles' role in diseases, but more research is needed on its connection to CKD and lifestyle factors. Therefore, we utilized the latest metabolomics data from the UK Biobank to explore the relationship between plasma metabolites and lifestyle factors, as well as to investigate the associations between various factors, including lifestyle-related metabolites, and the latent phase of CKD onset.
View Article and Find Full Text PDFOmega-3 fatty acids supplementation from late pregnancy to early lactation attenuates the endocannabinoid system and immune proteome in preovulatory follicles and endometrium of Holstein dairy cows.
J Dairy Sci
January 2025
Department of Ruminant Science, Institute of Animal Sciences, Agricultural Research Organization, Volcani Institute, Rishon LeZion, Israel. Electronic address:
Activation of the endocannabinoid system (ECS) elicits negative effects on the reproductive system in mammals. Omega-3 (n-3) fatty acid (FA) supplementation lowers ECS activation and has anti-inflammatory effects. Thus, we hypothesized that supplementing cows with n-3 FA will downregulate components of the ECS and immune system in preovulatory follicles and in the endometrium.
View Article and Find Full Text PDFPan-PPAR agonist lanifibranor improves insulin resistance and hepatic steatosis in patients with T2D and MASLD.
J Hepatol
January 2025
Division of Endocrinology, Diabetes and Metabolism, University of Florida, Gainesville, Florida, United States of America. Electronic address:
Background & Aims: Lanifibranor is a pan-PPAR agonist that improves glucose/lipid metabolism and reverses steatohepatitis and fibrosis in adults with MASH. We tested its effect on insulin resistance at the level of different target tissues in relationship to change in intrahepatic triglyceride (IHTG) content.
Methods: This phase 2, single center, study randomized (1:1) 38 patients with T2D and MASLD to receive lanifibranor 800 mg or placebo for 24 weeks.
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