Results 1 to 10 of about 13,000,271 (286)

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

The odds and implications of coinheritance of hemophilia A and B [PDF]

Research and Practice in Thrombosis and Haemostasis, 2020
We report 2 patients with coinheritance of the X‐linked bleeding disorders hemophilia A and hemophilia B. We describe the family pedigrees, clinical features, and genotyping.
Corinne Karch, Diane Masser‐Frye, Jacqueline Limjoco, Sarah E. Ryan, Shelley N. Fletcher, Kevin D. Corbett, Jill M. Johnsen, Courtney D. Thornburg   +7 more
doaj   +6 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

Carrier Detection in Hemophilia B. [PDF]

Thrombosis and Haemostasis, 1977
We examined 37 obligatory carriers of hemophilia B and 40 normal women. The levels of both factor IX activity and factor IX antigen were determined. The factor IX antigen levels were assayed in an inhibitor neutralization test in which a rabbit antiserum was used.
E Briët, Rogier M. Bertina, Nico H. van Tilburg, J.J. Veltkamp   +3 more
openalex   +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

Hemophilia B: A Pain in the Back. [PDF]

Cureus, 2023
This case report describes an unusual manifestation of hemophilia B, in the form of a lateral chest wall hematoma. A 27-year-old hemophiliac male was found to have a lateral chest wall hematoma after presenting with back pain associated with localized chest wall swelling.
Vedire A, Upadrasta G, Imburgio S, Johal AS, Hossain MA.   +4 more
europepmc   +3 more sources

The benefits of prophylaxis in patients with hemophilia B [PDF]

Expert Review of Hematology, 2018
Introduction: The health benefits of prophylactic dosing regimens for clotting factor therapy in patients with hemophilia include reduced joint damage and improved quality of life; as such, prophylaxis is recommended in treatment guidelines. However, many patients with hemophilia B are treated on demand, and prophylaxis has been utilized less ...
G. Castaman
openaire   +4 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

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