Results 31 to 40 of about 69,645 (303)

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors

New England Journal of Medicine, 2018
BACKGROUND Emicizumab is a bispecific monoclonal antibody that bridges activated factor IX and factor X to replace the function of missing activated factor VIII, thereby restoring hemostasis.
J. Mahlangu, J. Oldenburg, I. Paz‐Priel, C. Négrier, M. Niggli, E. Mancuso, C. Schmitt, V. Jiménez‐Yuste, C. Kempton, C. Dhalluin, M. Callaghan, W. Buján, M. Shima, J. Adamkewicz, E. Asikanius, G. Levy, R. Kruse-Jarres   +16 more
semanticscholar   +1 more source

MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA

Mediterranean Journal of Hematology and Infectious Diseases, 2020
The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino, Paul Tieu, Dr., Antony Chan   +2 more
doaj   +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

Clinical, instrumental, serological and histological findings suggest that hemophilia B may be less severe than hemophilia A

Haematologica, 2016
Recent evidence suggests that patients with severe hemophilia B may have a less severe disease compared to severe hemophilia A. To investigate clinical, radiological, laboratory and histological differences in the arthropathy of severe hemophilia A and ...
Daniela Melchiorre, Silvia Linari, Mirko Manetti, Eloisa Romano, Francesco Sofi, Marco Matucci-Cerinic, Christian Carulli, Massimo Innocenti, Lidia Ibba-Manneschi, Giancarlo Castaman   +9 more
doaj   +1 more source

Porcine model of hemophilia A. [PDF]

PLoS ONE, 2012
Hemophilia A is a common X chromosome-linked genetic bleeding disorder caused by abnormalities in the coagulation factor VIII gene (F8). Hemophilia A patients suffer from a bleeding diathesis, such as life-threatening bleeding in the brain and harmful ...
Yuji Kashiwakura, Jun Mimuro, Akira Onishi, Masaki Iwamoto, Seiji Madoiwa, Daiichiro Fuchimoto, Shunichi Suzuki, Misae Suzuki, Shoichiro Sembon, Akira Ishiwata, Atsushi Yasumoto, Asuka Sakata, Tsukasa Ohmori, Michiko Hashimoto, Satoko Yazaki, Yoichi Sakata   +15 more
doaj   +1 more source

Occurrence of FVIII Inhibitors in Hemophilia A Patients Following an Institutional Switch to a Third Generation B-Domain-Deleted FVIII

Clinical and Applied Thrombosis/Hemostasis, 2023
In 2018, Refacto AF R , a B-domain-deleted third-generation FVIII concentrate, became our preferential product. After the introduction, the development of inhibitors was prospectively monitored; retrospectively, we sought for risk factors in the patients
Louise H Hooimeijer MD, Marjet A Stein-Wit MD, Marja AJ Voskuilen RN, Michaël V Lukens PhD, Karina Meijer MD, PhD, Anja BU Mäkelburg MD, PhD, Rienk YJ Tamminga MD, PhD   +6 more
doaj   +1 more source

Current Situation and Prospect of Pharmacoeconomic Evaluation of Hemophilia in China

罕见病研究, 2022
In 2018, hemophilia, one of the first ones, entered into the catalogue of Rare Disease in China. However, drug accessibility and affordability have been the challenges for hemophilia patients.
LI Shunping, DOU Lei, FANG Yunhai
doaj   +1 more source

Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia

Journal of Clinical Medicine, 2021
Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible cure for hemophilia. However, immune responses directed against AAV vectors remain a hurdle to the broader use of this gene transfer platform.
P. Monahan, C. Négrier, M. Tarantino, L. Valentino, F. Mingozzi   +4 more
semanticscholar   +1 more source

Recurrent bleeding after rubber band ligation diagnosed as mild hemophilia B: a case report and literature review

BMC Surgery, 2022
Background Hemophilia is a recessive hemorrhagic disease relevant to X chromosome. In mild hemophilia cases, spontaneous bleeding is rare and the blood clotting function is normal, but severe bleeding may occur after trauma or surgery.
Xiaoying Jiang, Min Xu, Yaqing Ding, Yongqing Cao, Yibin Pan   +4 more
doaj   +1 more source

Establishment of Care System for Hemophilia in China: Current Status and Future Prospect

罕见病研究, 2022
Hemophilia is a X-linked recessive hereditary bleeding disorders. The patients need to receive replacement treatment with coagulation factors in their whole lives.
YANG Renchi
doaj   +1 more source