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| http://dx.doi.org/10.1007/s12630-020-01657-w | DOI Listing |
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Background: With the emergence of disease-modifying therapies (DMTs) for early Alzheimer's disease (AD), evolution of healthcare infrastructure is needed to identify patients eligible for treatment with an AD DMT, and to adequately treat and monitor patients throughout. Our research aimed to assess current practices and available infrastructure, to identify bottlenecks, and to propose solutions to increase patient access to AD DMTs in the EU4+UK.
Methods: We searched publicly available information (to August 2023) to derive a foundational understanding of the current level of healthcare infrastructure in each country.
The Metabolic Treatabolome and Inborn Errors of Metabolism Knowledgebase therapy tool: Do not miss the opportunity to treat!
J Inherit Metab Dis
January 2025
Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands.
Inborn errors of metabolism (IEMs) are rare genetic conditions with significant morbidity and mortality. Technological advances have increased therapeutic options, making it challenging to remain up to date. A centralized therapy knowledgebase is needed for early diagnosis and targeted treatment.
View Article and Find Full Text PDFRepurposing of Metformin to Improve Survival Outcomes in Patients With Upper Tract Urothelial Carcinoma.
Cancer Med
January 2025
Department of Urology, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan.
Purpose: Upper tract urothelial carcinoma (UTUC) presents a higher incidence rate in Taiwan compared to Western societies. The aim of this study is to investigate the potential of metformin in improving survival outcomes for patients with UTUC in Taiwan.
Material And Methods: This retrospective study included 940 patients with UTUC and type 2 diabetes from the Taiwan UTUC Collaboration Group, spanning 21 hospitals from July 1988 to September 2023.
Reusing zebrafish facility equipment: Planning, investment and what we have learned.
Lab Anim
December 2024
Instituto Murciano de Investigación Biomédica-Pascual Parrilla, Murcia, Spain.
The purpose of this study is to provide a detailed account of our successful experience in establishing a functional zebrafish holding facility by repurposing materials from a previous installation. On the eve of the start-up of our new animal facility we were notified that a research centre was putting part of its zebrafish holding facility (29 racks, accessories, water treatment unit) up for sale. Although the originally planned room was designed for six double racks, but encouraged by the increasing use of the zebrafish model, we decided to seize the opportunity, purchase the equipment and utilize it to create a larger configuration and an independent quarantine to protect the main facility.
View Article and Find Full Text PDFHereditary Hemorrhagic Telangiectasia: On the Brink of a New Treatment Era?
Semin Thromb Hemost
December 2024
Gastroenterology Department, HHT European Reference Center, ASST Ospedale Maggiore Crema, Crema, Italy.
Hereditary hemorrhagic telangiectasia (HHT) is an inherited vascular disorder with highly variable penetrance, affecting up to 1 in 5,000 individuals. It is characterized by the presence of abnormal blood vessels that can lead to excessive bleeding-most frequently recurrent nosebleeds (epistaxis), skin and mucosal telangiectasias (small, dilated blood vessels), as well as arteriovenous malformations (AVMs) that can form in various organs, particularly the lungs, liver, and brain. HHT is caused by loss-of-function mutations in the BMP9-10/ENG/ALK1/SMAD4 signaling pathway, an important mediator of vascular quiescence.
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