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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 PDFN7-methylguanosine modification in cancers: from mechanisms to therapeutic potential.
J Hematol Oncol
January 2025
Department of Gynecology, Xiangya Hospital, Central South University, Changsha, 410008, Hunan, China.
N7-methylguanosine (m7G) is an important RNA modification involved in epigenetic regulation that is commonly observed in both prokaryotic and eukaryotic organisms. Their influence on the synthesis and processing of messenger RNA, ribosomal RNA, and transfer RNA allows m7G modifications to affect diverse cellular, physiological, and pathological processes. m7G modifications are pivotal in human diseases, particularly cancer progression.
View Article and Find Full Text PDFEmerging strategies for nitric oxide production and their topical application as nanodressings to promote diabetic wound healing.
J Nanobiotechnology
January 2025
Department of Biobmedical Engineering and Technology, Institute of Basic Theory for Chinese Medicine, China Academy of Chinese Medical Sciences, Beijing, 100700, China.
The challenges associated with prolonged healing or non-healing of chronic diabetic wounds contribute significantly to the increased incidence of lower limb amputation. A pivotal factor in the impediment of healing is the reduced production of endogenous nitric oxide (NO) due to the hyperglycemic microenvironment typical of chronic diabetes. While both endogenous and exogenous NO have been shown to promote the healing process of diabetic wounds, the direct application of NO in wound management is limited due to its gaseous nature and the risk of explosive release.
View Article and Find Full Text PDFMolecular and cellular characteristics of cerebrovascular cell types and their contribution to neurodegenerative diseases.
Mol Neurodegener
January 2025
The Picower Institute for Learning and Memory, Cambridge, MA, USA.
Many diseases and disorders of the nervous system suffer from a lack of adequate therapeutics to halt or slow disease progression, and to this day, no cure exists for any of the fatal neurodegenerative diseases. In part this is due to the incredible diversity of cell types that comprise the brain, knowledge gaps in understanding basic mechanisms of disease, as well as a lack of reliable strategies for delivering new therapeutic modalities to affected areas. With the advent of single cell genomics, it is now possible to interrogate the molecular characteristics of diverse cell populations and their alterations in diseased states.
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