Results 11 to 20 of about 13,086,034 (342)
The odds and implications of coinheritance of hemophilia A and B [PDF]
Research and Practice in Thrombosis and Haemostasis, 2020 We report 2 patients with coinheritance of the X‐linked bleeding disorders hemophilia A and hemophilia B. We describe the family pedigrees, clinical features, and genotyping.
Corinne Karch +7 more
doaj +6 more sources
The benefits of prophylaxis in patients with hemophilia B [PDF]
Expert Review of Hematology, 2018 Introduction: The health benefits of prophylactic dosing regimens for clotting factor therapy in patients with hemophilia include reduced joint damage and improved quality of life; as such, prophylaxis is recommended in treatment guidelines.
G. Castaman
semanticscholar +4 more sources
Carrier Detection in Hemophilia B. [PDF]
Thrombosis and Haemostasis, 1977 We examined 37 obligatory carriers of hemophilia B and 40 normal women. The levels of both factor IX activity and factor IX antigen were determined. The factor IX antigen levels were assayed in an inhibitor neutralization test in which a rabbit antiserum was used.
E Briët +3 more
openalex +2 more sources
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
Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
New England Journal of Medicine, 2022 BACKGROUND FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B.
P. Chowdary +17 more
semanticscholar +1 more source
Managing Relevant Clinical Conditions of Hemophilia A/B Patients
Hematology Reports, 2023 The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini +9 more
doaj +1 more source
Current Medical Research and Opinion, 2022 Objective Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life.
Jinesh Shah +4 more
semanticscholar +1 more source
The Molecular Basis of FIX Deficiency in Hemophilia B
International Journal of Molecular Sciences, 2022 Coagulation factor IX (FIX) is a vitamin K dependent protein and its deficiency causes hemophilia B, an X-linked recessive bleeding disorder. More than 1000 mutations in the F9 gene have been identified in hemophilia B patients.
G. Shen, Meng Gao, Qing Cao, Weikai Li
semanticscholar +1 more source
Hemophilia B: A Pain in the Back. [PDF]
Cureus, 2023 This case report describes an unusual manifestation of hemophilia B, in the form of a lateral chest wall hematoma. A 27-year-old hemophiliac male was found to have a lateral chest wall hematoma after presenting with back pain associated with localized chest wall swelling.
Vedire A +4 more
europepmc +3 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