Results 21 to 30 of about 69,645 (303)
Establishment and Evolution of China National Hemophilia Registry
罕见病研究, 2022 Hemophilia is an inherited bleeding disorder and a type of rare disease that is hereditary, lifelong and disabling. The establishment of a National Hemophilia Registry is foundational to treating hemophilia.
XUE Feng, YANG Renchi
doaj +1 more source
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.
New England Journal of Medicine, 2020 BACKGROUND Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration of AAV5-hFVIII-SQ.
K. Pasi +10 more
semanticscholar +1 more source
Impact of novel hemophilia therapies around the world
Research and Practice in Thrombosis and Haemostasis, 2022 Hemophilia A and B are hereditary bleeding disorders, characterized by factor VIII or IX deficiencies, respectively. For many decades, prophylaxis with coagulation factor concentrates (replacement therapy) was the standard‐of‐care approach in hemophilia.
M. Ozelo, Gabriela G Yamaguti-Hayakawa
semanticscholar +1 more source
Development and Validation of a Liquid Chromatography High-Resolution Mass Spectrometry Method for Blood Desmopressin Quantification and Its Application in Hemophilia A Patients. [PDF]
Rapid Commun Mass SpectromABSTRACT Desmopressin (DDAVP), which indirectly increases Coagulation Factor VIII concentrations in the blood, is a common treatment for bleeding disorders such as von Willebrand disease or hemophilia A. However, DDAVP exhibits significant variability in response due to interindividual differences in pharmacokinetics.
Hodin S +7 more
europepmc +2 more sources
International recommendations on the diagnosis and treatment of acquired hemophilia A
Haematologica, 2020 Acquired hemophilia A (AHA), a rare bleeding disorder caused by neutralizing autoantibodies against coagulation factor VIII (FVIII), occurs in both men and women without a previous history of bleeding.
A. Tiede +11 more
semanticscholar +1 more source
Journal of Thrombosis and Haemostasis, 2021 Hemophilia A and B predominantly attracts clinical attention in males due to X‐linked inheritance, introducing a bias toward female carriers to be asymptomatic.
K. V. van Galen +11 more
semanticscholar +1 more source
Factor VIII and Factor IX Activity Measurements for Hemophilia Diagnosis and Related Treatments
Seminars in Thrombosis and Hemostasis, 2022 Accurate measurement of clotting factors VIII (FVIII) or IX (FIX) is vital for comprehensive diagnosis and management of patients with hemophilia A or B. The one-stage activated partial thromboplastin time (aPTT)-based clotting assay is the most commonly
A. Bowyer, R. Gosselin
semanticscholar +1 more source
Joint status of patients with nonsevere hemophilia A
Journal of Thrombosis and Haemostasis, 2022 Joint bleeding in hemophilia may eventually lead to joint damage. In nonsevere hemophilia, joint bleeds occur infrequently. Currently, knowledge on the joint status of patients with nonsevere hemophilia using objective imaging is limited.
A. Zwagemaker +11 more
semanticscholar +1 more source
Nature Biotechnology, 2020 Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine factor VIII (AAV-cFVIII) corrected the FVIII deficiency to 1.9–11.3% of ...
Giang N. Nguyen +14 more
semanticscholar +1 more source
Gene Therapy for Hemophilia: a review on clinical benefit, limitations and remaining issues.
Blood, 2021 In the past decade enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered AAV-based liver-directed gene therapy in patients with severe hemophilia B ...
F. Leebeek, Wolfgang A. Miesbach
semanticscholar +1 more source