Results 11 to 20 of about 82,032 (316)
Journal of Thrombosis and Haemostasis, 2010 Mild hemophilia A (HA), defined by clinical features and factor VIII coagulant activity (FVIII:C) between 0.05 and 0.40 IU mL(-1), is characteristically distinct from severe HA. Indeed, although the molecular characterization of mild HA has permitted the identification of specific underlying mutations, its clinical phenotype is strikingly different ...
Franchini M +2 more
openaire +2 more sources
Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy
Hepatology, EarlyView., 2022 Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann +25 more
wiley +1 more source
Baylor University Medical Center Proceedings, 2019 Acquired inhibitors of coagulation are a group of rare but potentially life-threatening blood disorders characterized by the presence of autoantibodies directed against clotting factor. Autoantibody against factor VIII is the most common form of clotting factor inhibitor, a condition also known as acquired hemophilia A.
Yadav, Pandey +4 more
openaire +2 more sources
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
New therapies for von Willebrand disease
Blood Advances, 2019 : The management of von Willebrand disease (VWD) is based upon the dual correction of the primary hemostasis defect, due to the inherited deficiency of von Willebrand factor (VWF), and of the secondary defect of factor VIII coagulant activity (FVIII:C ...
Pier Mannuccio Mannucci
doaj +1 more source
BMC Health Services Research, 2022 Background Hemophilia care in mainland China has been greatly improved since the establishment of the Hemophilia Treatment Center Collaborative Network of China (HTCCNC), and most of drugs for hemophilia have been covered by basic medical insurance ...
Zhengwei Huang +6 more
doaj +1 more source
The history and evolution of the clinical effectiveness of haemophilia type a treatment: a systematic review. [PDF]
, 2012 First evidence of cases of haemophilia dates from ancient Egypt, but it was when Queen Victoria from England in the 19th century transmitted this illness to her descendants, when it became known as the "royal disease".
A Aleem +48 more
core +2 more sources
Clinical and Biomedical Research, 2023 Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich +7 more
doaj
A Rare Case of Acquired Hemophilia A in Adolescents and Young Adults
Clinical Pediatric Hematology-Oncology, 2022 Acquired hemophilia A (AHA) is a rare bleeding disorder, especially in adolescents and young adults (AYAs) attributable to the development of autoantibodies against coagulation factor VIII (FVIII). AHA diagnosis is difficult; patients lack any history of
Min Jeong Lee, Young Shil Park
doaj +1 more source
Hemophilia: a biography on therapeutical approaches
Clinical and Biomedical Research, 2023 The history of hemophilia is ancient, with descriptions dated to the 2nd century AD. The first modern narratives appeared in 1800s, when total blood transfusion was the only available treatment and life expectancy was remarkably low.
Dayenne Catelli +7 more
doaj