Hematology, Transfusion and Cell Therapy, 2023 Objective: Factor X deficiency (FXd) is a rare coagulation disorder that can be either hereditary or acquired. Case report: We characterized patients with FXd and evaluated their bleeding patterns and treatment strategies. Methodology: This retrospective
Nigar Abdullayeva +3 more
doaj +1 more source
Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia [PDF]
, 2017 The most worrying complication of replacement therapy for severe hemophilia A and B is currently the occurrence of inhibitory alloantibodies against infused factor VIII and factor IX, respectively.
Franchini, Massimo +6 more
core +1 more source
Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A. [PDF]
, 2012 nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Abriss, Daniela +9 more
core +2 more sources
Current Situation and Prospect of Pharmacoeconomic Evaluation of Hemophilia in China
罕见病研究, 2022 In 2018, hemophilia, one of the first ones, entered into the catalogue of Rare Disease in China. However, drug accessibility and affordability have been the challenges for hemophilia patients.
LI Shunping, DOU Lei, FANG Yunhai
doaj +1 more source
Recombinant factorVIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A [PDF]
, 2015 This work was supported by funding from Biogen, including funding for the editorial and writing support in the the development of this ...
Allen, G +11 more
core +1 more source
A review of evidence about behavioural and psychological aspects of chronic joint pain among people with haemophilia [PDF]
, 2014 Joint pain related to haemophilia affects large numbers of people and has a significant impact on their quality of life. This article reviews evidence about behavioural and psychological aspects of joint pain in haemophilia, and considers that evidence ...
Acharya +75 more
core +2 more sources
MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA
Mediterranean Journal of Hematology and Infectious Diseases, 2020 The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino +2 more
doaj +1 more source
Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]
, 2001 Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A +5 more
core +1 more source
Haematologica, 2016 Recent evidence suggests that patients with severe hemophilia B may have a less severe disease compared to severe hemophilia A. To investigate clinical, radiological, laboratory and histological differences in the arthropathy of severe hemophilia A and ...
Daniela Melchiorre +9 more
doaj +1 more source
Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]
, 2002 The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Davidoff, AM +12 more
core +1 more source