Results 21 to 30 of about 22,643,381 (355)

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, P. Monahan, M. Eyster, S. Sullivan, M. Ragni, S. Croteau, J. Rasko, M. Recht, B. Samelson-Jones, A. Macdougall, Kristen Jaworski, R. Noble, M. Curran, K. Kuranda, F. Mingozzi, Tiffany Chang, K. Reape, X. Anguela, K. High   +18 more
semanticscholar   +1 more source

Updates on Novel Non-Replacement Drugs for Hemophilia

Pharmaceuticals, 2022
Over the last decade, the world of hemophilia has experienced an unprecedented therapeutic advance, thanks to the progress in bioengineering technologies, leading to the introduction of drugs with novel mechanisms of action based on restoring thrombin ...
Roberta Gualtierotti, Samantha Pasca, Alessandro Ciavarella, Sara Arcudi, Andrea Giachi, Isabella Garagiola, Chiara Suffritti, Simona Maria Siboni, Flora Peyvandi   +8 more
doaj   +1 more source

Surgical Procedures Requiring Hospitalization and Perioperative Management for Patients with Hereditary Bleeding Disorders

Clinical Pediatric Hematology-Oncology, 2022
Background : : Hemophilia requires a lifetime care for bleeding control and complications. Although patients diagnosed with hemophilia receive factor replacement, they also experience a variety of medical problems as they age.
Hyeun Su Seo, Won Kee Ahn, Seung Min Hahn, Jung Woo Han, Chuhl Joo Lyu   +4 more
doaj   +1 more source

Immunogenicity of Current and New Therapies for Hemophilia A

Pharmaceuticals, 2022
Anti-drug antibody (ADA) development is a significant complication in the treatment of several conditions. For decades, the mainstay of hemophilia A treatment was the replacement of deficient coagulation factor VIII (FVIII) to restore hemostasis, control,
A. Prezotti, Jéssica O Frade-Guanaes, Gabriela G Yamaguti-Hayakawa, M. Ozelo   +3 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

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

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, T. Lissitchkov, V. Mamonov, T. Mant, M. Timofeeva, C. Bagot, P. Chowdary, P. Georgiev, L. Gercheva‐Kyuchukova, Kate Madigan, H. Nguyen, Qifeng Yu, B. Mei, C. Benson, M. Ragni   +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, Fabienne R Kloosterman, R. Hemke, S. Gouw, M. Coppens, Lorenzo G R Romano, M. Kruip, M. Cnossen, F. Leebeek, B. Hutten, M. Maas, K. Fijnvandraat   +11 more
semanticscholar   +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, S. Rangarajan, N. Mitchell, W. Lester, E. Symington, B. Madan, M. Laffan, C. Russell, Mingjin Li, Glenn F Pierce, W. Y. Wong   +10 more
semanticscholar   +1 more source

A factor VIIIa–mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice

Blood, 2021
Østergaard and colleagues report the development of a potent bispecific antibody, Mim8, which binds both activated factor IX and factor X, mimicking the propagation of coagulation by activated factor VIII.
Henrik Østergaard, J. Lund, Per Greisen, S. Kjellev, A. Henriksen, Nikolai Lorenzen, E. Johansson, G. Røder, M. G. Rasch, L. Johnsen, T. Egebjerg, S. Lund, H. Rahbek-Nielsen, Prafull S Gandhi, K. Lamberth, M. Loftager, L. Andersen, A. C. Bonde, F. Stavenuiter, D. E. Madsen, Xun Li, T. L. Holm, C. D. Ley, P. Thygesen, Haisun Zhu, Rong Zhou, Karina Thorn, Zhiru Yang, M. B. Hermit, J. Bjelke, B. G. Hansen, I. Hilden   +31 more
semanticscholar   +1 more source

Emicizumab initiation and bleeding outcomes in people with hemophilia A with and without inhibitors: A single‐center report

Research and Practice in Thrombosis and Haemostasis, 2021
Background Emicizumab, a bispecific antibody factor VIII mimetic, is approved for prophylaxis in hemophilia, and has different risks and side effects compared to factor VIII products.
Beth Boulden Warren, Adrian Chan, Marilyn Manco‐Johnson, Brian R. Branchford, Tyler W. Buckner, Genevieve Moyer, Elizabeth Gibson, Dianne Thornhill, Michael Wang, Christopher J Ng   +9 more
doaj   +1 more source