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Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials.
Adv Ther
January 2025
Cytel, Inc., Waltham, MA, USA.
Introduction: Fabry disease (FD) is a rare lysosomal storage disorder that is associated with pain and progressive damage to the renal, cardiac, and cerebrovascular systems. Enzyme replacement therapy (ERT) is one of the treatment options for FD and the most recently approved ERT agent, pegunigalsidase alfa, has shown clinical efficacy in three phase 3 clinical trials of adults with FD: BALANCE, BRIDGE, and BRIGHT. Recent published guidelines support the mapping of health utility state data to the EuroQol-5 Dimension-3 Level (EQ-5D-3L) index to align with the preferred methodology used by the National Institute for Health and Care Excellence (NICE).
View Article and Find Full Text PDFHigh-Throughput Chemotherapeutic Drug Screening System for Gastric Cancer (Cure-GA).
Ann Surg Oncol
January 2025
Department of Surgery, Seoul National University Bundang Hospital, Seongnam, Republic of Korea.
Background: Three dimensional (3D) cell cultures can be effectively used for drug discovery and development but there are still challenges in their general application to high-throughput screening. In this study, we developed a novel high-throughput chemotherapeutic 3D drug screening system for gastric cancer, named 'Cure-GA', to discover clinically applicable anticancer drugs and predict therapeutic responses.
Methods: Primary cancer cells were isolated from 143 fresh surgical specimens by enzymatic treatment.
The Multimorbidity Questionnaire (MMQ1): English translation and validation of a Danish patient reported outcome measure for quality of life in people with multiple long-term conditions in a cross-sectional survey.
Qual Life Res
January 2025
Centre of General Practice, Department of Public Health, Faculty of Health Sciences, University of Copenhagen, Copenhagen, Denmark.
Purpose: MMQ1 is a Danish-language patient-reported outcome measure (PROM) for quality of life (QOL) in people with multiple long-term conditions (MLTC). It measures needs-based QOL across six scales: Physical ability, Concerns and worries, Limitations in daily life, Social life, Personal finances and Self-image. There is currently no such measure available in English.
View Article and Find Full Text PDFFasting plasma methylglyoxal concentrations are associated with higher numbers of circulating intermediate and non-classical monocytes but with lower activation of intermediate monocytes: the Maastricht Study.
J Endocrinol Invest
January 2025
Department of Internal Medicine, Maastricht University Medical Center, Maastricht, 6229ER, the Netherlands.
Purpose: Elevated methylglyoxal (MGO) levels and altered immune cell responses are observed in diabetes. MGO is thought to modulate immune cell activation. The current study investigated whether fasting or post-glucose-load plasma MGO concentrations are associated with circulating immune cell counts and activation in a large cohort study.
View Article and Find Full Text PDFReal-Life Experience with Tildrakizumab in Plaque Psoriasis with Palmoplantar Involvement: A Multi-Center Retrospective Italian Study.
Dermatol Ther (Heidelb)
January 2025
Department of Clinical and Molecular Sciences-Dermatological Clinic, Università Politecnica Delle Marche, Ancona, Italy.
Introduction: Palmoplantar psoriasis (PPp) has a profound negative impact on patients' quality of life, and it represents a therapeutic challenge, as palms and soles are difficult to treat area. Although the efficacy profile of tildrakizumab has been well evaluated in the literature, data on its use for PPp are still limited. The objective of the study was to evaluate the efficacy and safety of tildrakizumab on moderate-to-severe plaque psoriasis with involvement of the palmoplantar area.
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