Results 11 to 20 of about 22,597,991 (289)
Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
New England Journal of Medicine, 2023 BACKGROUND Efanesoctocog alfa provides high sustained factor VIII activity by overcoming the von Willebrand factor-imposed half-life ceiling. The efficacy, safety, and pharmacokinetics of efanesoctocog alfa for prophylaxis and treatment of bleeding ...
Annette von Drygalski +14 more
semanticscholar +1 more source
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
New England Journal of Medicine, 2023 BACKGROUND Valoctocogene roxaparvovec delivers a B-domain-deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A.
J. Mahlangu +28 more
semanticscholar +1 more source
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
New England Journal of Medicine, 2022 BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
M. Ozelo +22 more
semanticscholar +1 more source
Science Advances, 2022 Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic ...
J. Han +18 more
semanticscholar +1 more source
The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B
Blood Advances, 2018 : Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
J. Michael Soucie +4 more
doaj +2 more sources
Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)
Research and Practice in Thrombosis and Haemostasis, 2022 Emicizumab is a subcutaneously administered humanized, bispecific, monoclonal antibody approved for prophylaxis in people with hemophilia A.
Renchi Yang +11 more
semanticscholar +1 more source
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
New England Journal of Medicine, 2021 BACKGROUND The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose.
Lindsey A. George +18 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
Journal of Thrombosis and Haemostasis, 2021 Fitusiran, an investigational small interfering RNA therapy, reduces antithrombin production to rebalance hemostasis in people with hemophilia A or B, with or without inhibitors.
K. Pasi +14 more
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