Results 91 to 100 of about 12,392,515 (266)
Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
Journal of Translational Medicine, 2017 Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia.
David M. Markusic +8 more
doaj +1 more source
Depression and anxiety in people with hemophilia A and B
, 2023 Objective This study described the prevalence of and correlates of depression and anxiety in adult patients with hemophilia A and B. Methods In this cross-sectional study, we investigated patients with hemophilia who were being seen at an adult ...
Ozen Onen-Sertoz +7 more
core +1 more source
Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community
Haemophilia, EarlyView.ABSTRACT Inherited bleeding disorders encompass a diverse group of conditions caused by genetic defects affecting coagulation factors, fibrinogen, von Willebrand factor, or platelet function. Despite major advances in quantitative and functional laboratory assays, a substantial diagnostic gap remains, particularly in patients with mild or atypical ...
Anna R. Blankstein +6 more
wiley +1 more source
Patient Related Outcome Measures, 2019 Susan Cutter,1 Christine Guelcher,2 Susan Hunter,3 Dawn Rotellini,4 Spencer Dunn,5 David L Cooper61Penn Comprehensive Hemophilia and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA, USA; 2Comprehensive Hemostasis and ...
Cutter S +5 more
doaj
New developments in the management of moderate-to-severe hemophilia B
, 2016 Moniba Nazeef,1,2 John P Sheehan1,2 1Department of Medicine, Division of Hematology/Oncology, 2UW Carbone Cancer Center, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA Abstract: Hemophilia B is an X-linked genetic ...
Nazeef M, Sheehan JP
core
Haemophilia, EarlyView.ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini +4 more
wiley +1 more source
Hemophilia is a collection of hereditary bleeding disorders that are X-linked recessive. Patients often experience recurrent bleeding in joints, leading to a cascade of complications including chronic synovitis, hemophilic arthropathy, muscle atrophy ...
Hemophilia Treatment Center Collaborative Network of China
core +1 more source
Patient Preferences for the Treatment of Haemophilia A and B in France
Haemophilia, EarlyView.ABSTRACT Introduction Haemophilia impacts health and quality of life. Advances in treatments such as factor replacement therapy (FRT), bispecific monoclonal antibody (BS‐mAb), gene therapy (GT), rebalancing treatment and high sustained factor necessitate understanding patient preferences to inform therapeutic strategies. Aim This study quantified trade‐
Yesim Dargaud +10 more
wiley +1 more source
The spectrum of bleeding in women and girls with haemophilia B
, 2017 Although hemophilia B affects 1 in 25,000 males there may be 3 female hemophilia B carriers per affected male. This clinical review highlights the unique challenges faced by hemophilia B carriers including the under‐recognition of bleeding symptoms ...
Grabowski, E. F +11 more
core +1 more source
Haemophilia, EarlyView.ABSTRACT Introduction Efficacy and safety of albutrepenonacog alfa (rIX‐FP) in people with haemophilia B (PwHB) has been previously demonstrated in clinical trials. However, real‐world data insights are required. Aim To describe real‐world effectiveness and tolerability of rIX‐FP in PwHB in Germany.
Johannes Oldenburg +9 more
wiley +1 more source