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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 PDFLangerhans cell histiocytosis in children: the value of ultrasound in diagnosis and follow-up.
BMC Med Imaging
January 2025
Department of Ultrasound Medicine, First Affiliated Hospital, Fujian Medical University, Fuzhou, 350005, China.
Background: Langerhans cell histiocytosis (LCH) is a rare disease, most prevalent in children. Ultrasound is a noninvasive, cheap, and widely available technique. However, systematic elucidation of sonographic features of LCH and treatment related follow-up are relatively few, resulting in overall underestimation of the clinical value of ultrasound in diagnosing and monitoring LCH.
View Article and Find Full Text PDF"Evaluation of Curcuma zedoaria Rosc. in the management of non-alcoholic fatty liver Disease: A Randomized, single blind, controlled trial".
Arab J Gastroenterol
January 2025
PG Dept. of Moalajat, (Medicine) RRIUM, Srinagar, India.
Background And Study Aims: Non-alcoholic fatty liver disease (NAFLD) is the most prevalent chronic liver disorder, affecting 23% to 32% of the global population. This clinical study aimed to assess the efficacy of Curcuma zedoaria Rosc. compared to vitamin E in managing NAFLD.
View Article and Find Full Text PDFWe created and validated the Neuro-Score, a specific scale to detect and monitor cognitive impairment, including mild stages, in kidney or liver transplant recipients. A qualitative study was conducted to define a preliminary set of 62 items. Item reduction was performed using exploratory factor analysis.
View Article and Find Full Text PDFBaseline Alpha-Fetoprotein Elevation and the Risk of Hepatocellular Carcinoma in Chronic Hepatitis B: A Multicentre Cohort Study.
J Viral Hepat
March 2025
Department of Biomedical Informatics, Ajou University School of Medicine, Suwon, Republic of Korea.
Alpha-fetoprotein (AFP) level and its changes in chronic hepatitis B (CHB) may influence the risk of future hepatocellular carcinoma (HCC). This study aims to evaluate the HCC risk in CHB patients with no overt HCC but with elevated AFP level and to explore the prognostic role of longitudinal changes in AFP and liver-related laboratory values. This multicentre cohort study included 10,639 CHB patients without a history of HCC from seven medical facilities in South Korea.
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