Results 11 to 20 of about 31,863 (238)
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
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
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
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
Journal of Blood Medicine, 2022 Randall Curtis,1 Marilyn Manco-Johnson,2 Barbara A Konkle,3 Roshni Kulkarni,4 Joanne Wu,5 Judith R Baker,6 Megan Ullman,7 Duc Quang Tran Jr,8 Michael B Nichol5 1Factor VIII Computing, Berkeley, CA, USA; 2Hemophilia and Thrombosis Center, University of ...
Curtis R +8 more
doaj
Practices and challenges for hemophilia management under resource constraints in Thailand
Orphanet Journal of Rare Diseases, 2023 Hemophilia is an inherited bleeding disorder caused by deficiency of a specific coagulation factor. Factor VIII deficiency is responsible for hemophilia A while factor IX deficiency is responsible for hemophilia B. As per the 2020 annual global survey by
Chatphatai Moonla +7 more
doaj +1 more source
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
"Prevalence of inhibitors in a population of 1280 Hemophilia A patients in Iran " [PDF]
Acta Medica Iranica, 2003 Development of inhibitor to factor VIII is the most serious complication of hemophilia therapy. To determine the prevalence of inhibitors in Iran hemophilia A patients exposed to blood products, 1280 hemophilia A patients (age range 9 months-84 years ...
Sharifian R +6 more
doaj +2 more sources
Hemophilia management: Huge impact of a tiny difference
Research and Practice in Thrombosis and Haemostasis, 2020 Hemophilia A and B are inherited X‐linked disorders of hemostasis, associated with an increased bleeding tendency. Patients with severe hemophilia have undetectable clotting factor levels and experience spontaneous bleeds.
Fabienne Kloosterman +5 more
doaj +1 more source
Veterinary Sciences, 2022 A ten-week-old male Golden retriever puppy was presented with severe hyperesthesia, mild neurological deficits and episcleral bleeding. Clotting times showed a normal prothrombin time (PT) and prolonged activated partial thromboplastin time (aPPT ...
Charlotte Lubbers +3 more
doaj +1 more source