Results 1 to 10 of about 49,324 (252)

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

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

<i>F9</i> missense variant hot spots associated with qualitative protein defects causing hemophilia B. [PDF]

Blood Vessel Thromb Hemost
van Duijl TT, Gouw S, Kronevska I, Kooiker A, Kopatz WF, Freato N, Beijlevelt M, Hamer HM, Fijnvandraat K, Meijers JCM, van den Biggelaar M.   +10 more
europepmc   +2 more sources

ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B. [PDF]

Eur J Haematol
Volot F, Castet S, Fournel A, Frotscher B, Gillet B, Desprez D, Tardy B, Rauch A, Chamouni P, Biron-Andreani C, Valentin JB, Harroche A, Dargaud Y, Petesch BP, d'Oiron R, Berger C, Reynes C, Lauvray T, de Raucourt E, Hassoun A, Lebreton A, Cussac V, Catovic H, Martin C, Guillet B.   +24 more
europepmc   +3 more sources

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

Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study. [PDF]

Mol Ther Methods Clin Dev
Klamroth R, Recht M, Key NS, Miesbach W, Pipe SW, Kaczmarek R, Drelich D, Salazar B, Le Quellec S, Monahan PE, Galante N, van der Valk P, Tarrant J.   +12 more
europepmc   +2 more sources

Managing Relevant Clinical Conditions of Hemophilia A/B Patients

Hematology Reports, 2023
The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini, Jacopo Agnelli Giacchiello, Erminia Baldacci, Christian Carulli, Giancarlo Castaman, Anna Chiara Giuffrida, Giuseppe Malcangi, Angiola Rocino, Sergio Siragusa, Ezio Zanon   +9 more
doaj   +1 more source

Seroprevalence to adeno‐associated virus type 6 in people with hemophilia B from a UK adult cohort

Research and Practice in Thrombosis and Haemostasis, 2022
Background Gene therapy shows promise as a potential “cure” for hemophilia A and B. Adeno‐associated virus (AAV) vectors are the leading platform to deliver modified genetic code of factor VIII or IX to the liver effecting endogenous production.
Sara Boyce, Izabela James, Savita Rangarajan, Nicola Curry, Catherine Bagot, Steven Austin, Mike Laffan, Sarah Mangles, Kandiah Chandrakumaran, Carina Mundy   +9 more
doaj   +1 more source

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   +1 more source