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Evaluation of Lyso-Gb1 as a biomarker for Gaucher disease treatment outcomes using data from the Gaucher Outcome Survey.
Orphanet J Rare Dis
January 2025
Department of Gastroenterology, Hepatology and Infectious Diseases, University Hospital, Heinrich- Heine University, Düsseldorf, Germany.
Background: Patients with Gaucher disease (GD) require continual monitoring; however, lack of specific disease biomarkers was a significant challenge in the past. Glucosylsphingosine (lyso-Gb1) has been shown to be a reliable, key, specific, and sensitive biomarker for diagnosis, prognosis, and treatment response in clinical studies of patients with GD. We evaluated the change in lyso-Gb1 concentration over time following enzyme replacement therapy in patients with confirmed GD using real-world data from the Gaucher Outcome Survey disease registry.
View Article and Find Full Text PDFUnveiling new therapeutic horizons in rheumatoid arthritis: an In-depth exploration of circular RNAs derived from plasma exosomes.
J Orthop Surg Res
January 2025
Department of Rheumatology and Immunology, Affiliated Hospital of Yangzhou University, Yangzhou University, No. 368 Hanjiang Middle Road, Yangzhou, Jiangsu, 225000, China.
Rheumatoid arthritis (RA), a chronic inflammatory joint disease causing permanent disability, involves exosomes, nanosized mammalian extracellular particles. Circular RNA (circRNA) serves as a biomarker in RA blood samples. This research screened differentially expressed circRNAs in RA patient plasma exosomes for novel diagnostic biomarkers.
View Article and Find Full Text PDFValidating a clinically based MS-MLPA threshold through comparison with Sanger sequencing in glioblastoma patients.
Clin Epigenetics
January 2025
Center of Oncocytogenomics, Institute of Medical Biochemistry and Laboratory Diagnostics, General University Hospital and 1st Faculty of Medicine of Charles University in Prague, U Nemocnice 499/2, 128 00, Prague, Czech Republic.
Background: Glioblastoma is the commonest malignant brain tumor and has a very poor prognosis. Reduced expression of the MGMT gene (10q26.3), influenced primarily by the methylation of two differentially methylated regions (DMR1 and DMR2), is associated with a good response to temozolomide treatment.
View Article and Find Full Text PDFImpact of commercial gut health interventions on caecal metagenome and broiler performance.
Microbiome
January 2025
Faculty of Infectious and Tropical Diseases, London School of Hygiene and Tropical Medicine, London, UK.
Background: Maintaining gut health is a persistent and unresolved challenge in the poultry industry. Given the critical role of gut health in chicken performance and welfare, there is a pressing need to identify effective gut health intervention (GHI) strategies to ensure optimal outcomes in poultry farming. In this study, across three broiler production cycles, we compared the metagenomes and performance of broilers provided with ionophores (as the control group) against birds subjected to five different GHI combinations involving vaccination, probiotics, prebiotics, essential oils, and reduction of ionophore use.
View Article and Find Full Text PDFImpact of hospital formulary intervention on carbapenem use: a segmented time-series analysis of consumption and a propensity score-matched non-inferiority study of treatment efficacy.
J Pharm Health Care Sci
January 2025
Laboratory of Clinical Pharmacy Education, Research and Education Center for Clinical Pharmacy, School of Pharmacy, Kitasato University, 5-9-1, Shirokane, Minato-ku, Tokyo, 108-8641, Japan.
Background: Pharmaceutical formularies play a crucial role in guiding medication use by balancing clinical effectiveness and cost efficiency. Although formulary implementation has been increasing in Japan, comprehensive evaluations of its impact on both clinical and economic outcomes are limited. This study aimed to assess the effect of introducing an antimicrobial formulary at Yokohama City University Hospital on antibiotic usage and treatment outcomes in intra-abdominal infections.
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