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Serum KNG and FVIII may serve as potential biomarkers for depression.
Behav Brain Res
January 2025
Department of Psychiatry, Renmin Hospital of Wuhan University, Wuhan 430060, Hubei, PR China; Department of Psychiatry and Institute of Neuropsychiatry, Renmin Hospital of Wuhan University, Wuhan 430060, Hubei, PR China; Taikang Center for Life and Medical Sciences, Wuhan University, Wuhan, 430071, China. Electronic address:
Background: The global burden of major depressive disorder (MDD) is rising, with current diagnostic methods hindered by significant subjectivity and low inter-rater reliability. Several studies have implied underlying link between coagulation-related proteins, such as kininogen (KNG) and coagulation factor VIII (FVIII), and depressive symptoms, offering new insights into the exploration of depression biomarkers. This study aims to elucidate the roles of KNG and FVIII in depression, potentially providing a foundational basis for biomarker research in this field.
View Article and Find Full Text PDFFlow Cytometry Evaluation of Blood-Cell-Bound Surface FVIII in Hemophilia A and Thrombosis.
Cells
January 2025
Research Department, Sidra Medicine, Doha P.O. Box 26999, Qatar.
Hemophilia A (HA) is associated with FVIII coagulation insufficiency or inactivity leading to excessive bleeding. Elevated FVIII, on the contrary, is associated with thrombophilia, thrombosis, myocardial infarctions, and stroke. Active FVIII (aFVIII) uses its C2 domain to bind to blood cells' membranes, consequently carrying out its coagulative function.
View Article and Find Full Text PDFSafety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study.
J Thromb Haemost
January 2025
BioMarin Pharmaceutical Inc., Novato, CA, USA.
Background: Valoctocogene roxaparvovec, an adeno-associated virus vector that transfers a human factor VIII (FVIII) coding sequence to hepatocytes, provides bleeding protection for people with severe hemophilia A (HA).
Objective: Determine the efficacy and safety of valoctocogene roxaparvovec with concomitant prophylactic glucocorticoids in the open-label, single-arm, phase 3b GENEr8-3 trial.
Methods: Participants with severe HA who were using HA prophylaxis received one 6x10 vg/kg infusion of valoctocogene roxaparvovec concomitantly with daily prophylactic glucocorticoids (40 mg prednisolone equivalent/d weeks 0‒8; taper to 5 mg/d weeks 9‒19).
A phase 1/2 safety and efficacy study of TAK-754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials.
Haemophilia
December 2024
Investigative Toxicology, Takeda Development Center of the Americas, Cambridge, USA.
Introduction: Haemophilia A is an X-linked bleeding disorder resulting from a deficiency of factor VIII (FVIII). To date, multiple gene therapies have entered clinical trials with the goal of providing durable haemostatic protection from a single dose. TAK 754 (BAX 888) is an investigational AAV8-based gene therapy containing a FVIII transgene.
View Article and Find Full Text PDFEfficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial.
Res Pract Thromb Haemost
November 2024
Hemocentro University of Campinas, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil.
Background: Valoctocogene roxaparvovec, an adeno-associated virus-mediated gene therapy for severe hemophilia A, enables endogenous factor (F)VIII expression and provides bleed protection.
Objectives: Determine valoctocogene roxaparvovec durability, efficacy, and safety 4 years after treatment.
Methods: In the phase 3 GENEr8-1 trial, 134 adult male persons with severe hemophilia A without inhibitors and previously using FVIII prophylaxis received a 6 × 10 vg/kg infusion of valoctocogene roxaparvovec.
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