Results 1 to 10 of about 13,086,034 (342)

Gene Therapy Approaches for the Treatment of Hemophilia B. [PDF]

Int J Mol Sci, 2023
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14 days to 2–3 times a week in patients with severe hemophilia B, as a result of a single injection, gene therapy can restore F9 gene expression and maintain it for a ...
Soroka AB, Feoktistova SG, Mityaeva ON, Volchkov PY.   +3 more
europepmc   +2 more sources

CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs

Haematologica, 2020
Hemophilia B is an X-linked recessive bleeding disorder caused by abnormalities in the coagulation factor IX gene. Without prophylactic treatment, patients experience frequent spontaneous bleeding episodes.
Jiahuan Chen, Beiying An, Biao Yu, Xiaohuan Peng, Hongming Yuan, Qiangbing Yang, Xue Chen, Tingting Yu, Lingyu Wang, Xinwei Zhang, He Wang, Xiaodong Zou, Daxin Pang, Hongsheng Ouyang, Xiaochun Tang   +14 more
doaj   +2 more sources

Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A [PDF]

Journal of Thrombosis and Haemostasis, 2015
This work was supported by funding from Biogen, including funding for the editorial and writing support in the the development of this ...
Allen, G, Barnes, C, Cristiano, LM, Gambino, G, Kulkarni, R, Liesner, R, Mahlangu, J, Neelakantan, S, Nolan, B, Pasi, J, Pierce, GF, Young, G   +11 more
exaly   +3 more sources

Assessing the health-state utility values of rare disease-hemophilia B using EQ-5D-5L: a study based on the Chinese population [PDF]

Orphanet Journal of Rare Diseases
Background Obtaining health-state utility values (HSUVs) aids in making scientific decisions in patient health management, especially for rare disease patients.
Chuchuan Wan, Haotao Li, Yulin Zhang, Qiqi Wang, Yiwen Huang, Tao Guan, Xiaoyu Xi   +6 more
doaj   +2 more sources

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Orphanet Journal of Rare Diseases, 2021
Background Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life.
Tom Burke, Sohaib Asghar, Jamie O’Hara, Eileen K. Sawyer, Nanxin Li   +4 more
doaj   +2 more sources

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   +2 more sources

Total Hip Arthroplasty for Avascular Necrosis in a Patient With Hemophilia B [PDF]

Arthroplasty Today
Avascular necrosis (AVN) of the femoral head accounts for up to 10% of all total hip arthroplasties performed annually. Typically associated with intravascular coagulation, AVN is extremely rare in patients with bleeding disorders such as hemophilia B ...
Siddhartha Dandamudi, BBA, Joyee Tseng, BS, John Ratz, BS, Lisa Boggio, MD, MS, Brett R. Levine, MD, MS   +4 more
doaj   +2 more sources

Hemophilia B: molecular pathogenesis and mutation analysis

Journal of Thrombosis and Haemostasis, 2015
Hemophilia B is an X‐chromosome‐linked inherited bleeding disorder primarily affecting males, but those carrier females with reduced factor IX activity (FIX:C) levels may also experience some bleeding.
A. Goodeve
exaly   +2 more sources

Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 [PDF]

Journal of Controlled Release, 2019
Curiel, David T, Kashentseva, Elena, Lauron, Elvin J, Lu, Zhi Hong, Stephens, Calvin J, Yokoyama, Wayne M   +5 more
core   +3 more sources

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.

New England Journal of Medicine, 2023
BACKGROUND Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against bleeding ...
S. Pipe, F. Leebeek, M. Recht, N. Key, G. Castaman, Wolfgang A. Miesbach, S. Lattimore, K. Peerlinck, P. R. van der Valk, M. Coppens, P. Kampmann, K. Meijer, Niamh O'Connell, K. Pasi, D. Hart, R. Kazmi, J. Astermark, C. Hermans, R. Klamroth, Richard W. Lemons, N. Visweshwar, Annette von Drygalski, G. Young, S. Crary, M. Escobar, E. Gomez, R. Kruse-Jarres, D. Quon, E. Symington, Michael Wang, Allison P. Wheeler, R. Gut, Yinyin Liu, R. Dolmetsch, D. Cooper, Yanyan Li, B. Goldstein, P. Monahan   +37 more
semanticscholar   +1 more source