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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 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.
View Article and Find Full Text PDFUnderstanding retinal tau pathology through functional 2D and 3D iPSC-derived in vitro retinal models.
Acta Neuropathol Commun
January 2025
Department of Physiology and Pharmacology, Sapienza University of Rome, 00185, Rome, Italy.
The generation of retinal models from human induced pluripotent stem cells holds significant potential for advancing our understanding of retinal development, neurodegeneration, and the in vitro modeling of neurodegenerative disorders. The retina, as an accessible part of the central nervous system, offers a unique window into these processes, making it invaluable for both study and early diagnosis. This study investigates the impact of the Frontotemporal Dementia-linked IVS 10 + 16 MAPT mutation on retinal development and function using 2D and 3D retinal models derived from human induced pluripotent stem cells.
View Article and Find Full Text PDFFrom micro to macro, nanotechnology demystifies acute pancreatitis: a new generation of treatment options emerges.
J Nanobiotechnology
January 2025
Department of Gastroenterology, Shanghai Institute of Pancreatic Diseases, National Key Laboratory of Immunity and Inflammation, Changhai Hospital, Naval Medical University, Shanghai, 200433, China.
Acute pancreatitis (AP) is a disease characterized by an acute inflammatory response in the pancreas. This is caused by the abnormal activation of pancreatic enzymes by a variety of etiologic factors, which results in a localized inflammatory response. The symptoms of this disease include abdominal pain, nausea and vomiting and fever.
View Article and Find Full Text PDFConstruct validity and responsiveness of clinical upper limb measures and sensor-based arm use within the first year after stroke: a longitudinal cohort study.
J Neuroeng Rehabil
January 2025
Luzerner Kantonsspital, University, Teaching and Research Hospital, University of Lucerne, Lucerne, Switzerland.
Background: Construct validity and responsiveness of upper limb outcome measures are essential to interpret motor recovery poststroke. Evaluating the associations between clinical upper limb measures and sensor-based arm use (AU) fosters a coherent understanding of motor recovery. Defining sensor-based AU metrics for intentional upper limb movements could be crucial in mitigating bias from walking-related activities.
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