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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.

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Background: The events of October 7, 2023, and the subsequent war have starkly exposed the shortcoming of Israel's public mental health system. This system, already strained by years of underfunding and the COVID-19 pandemic, was unprepared for the surge in mental health needs resulting from these traumatic events. This paper outlines the systemic failures and proposes a comprehensive overhaul reform towards an integrative community-based, recovery-oriented mental health service.

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Impact 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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Background: One of the best ways to impart important skills to trainees is through simulation-based training, which is more reliable than the conventional clinical examination method. It is used in pediatric nursing training to allow nurses to practice and improve their clinical and conversational skills during an actual child encounter. A heel-prick is a complex psychomotor task that requires skill and knowledge from the pediatric nurse performing the procedure while applying for the National Newborn Screening Program.

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Background: Silicosis remains a major occupational health challenge in India. This review systematically examines the prevalence, risk factors, regional differences, and diagnostic tools specific to India's high-risk industries. Additionally, it assesses policy gaps and offers insights from diverse clinical and qualitative studies, aiming to inform targeted public health interventions and support the development of effective occupational health policies.

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