Results 1 to 10 of about 13,148,245 (315)

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

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

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   +3 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

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

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, W. 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, A. 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

Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.

New England Journal of Medicine, 2022
BACKGROUND FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B.
P. Chowdary, S. Shapiro, M. Makris, G. Evans, S. Boyce, K. Talks, G. Dolan, U. Reiss, M. Phillips, A. Riddell, M. R. Peralta, M. Quaye, D. W. Patch, E. Tuddenham, A. Dane, M. Watissée, A. Long, A. Nathwani   +17 more
semanticscholar   +1 more source

The Molecular Basis of FIX Deficiency in Hemophilia B

International Journal of Molecular Sciences, 2022
Coagulation factor IX (FIX) is a vitamin K dependent protein and its deficiency causes hemophilia B, an X-linked recessive bleeding disorder. More than 1000 mutations in the F9 gene have been identified in hemophilia B patients.
G. Shen, Meng Gao, Qing Cao, Weikai Li
semanticscholar   +1 more source

Medical costs and hospital utilization for hemophilia A and B urban inpatients in China: a national cross-sectional study

BMC Health Services Research, 2022
Background Hemophilia care in mainland China has been greatly improved since the establishment of the Hemophilia Treatment Center Collaborative Network of China (HTCCNC), and most of drugs for hemophilia have been covered by basic medical insurance ...
Zhengwei Huang, Stephen Nicholas, Yong Yang, Xiaoping Chen, Elizabeth Maitland, Yong Ma, Xuefeng Shi   +6 more
doaj   +1 more source

Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Current Medical Research and Opinion, 2022
Objective Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life.
Jinesh Shah, Hongseok Kim, K. Sivamurthy, P. Monahan, Michael Fries   +4 more
semanticscholar   +1 more source