Results 11 to 20 of about 113,823 (359)
Personalised Prophylaxis in a Child with Haemophilia A and Type 1 Diabetes
Clinics and Practice, 2021 Poor management of either type 1 diabetes or haemophilia A can lead to complications such as organ dysfunction and haemarthropathy. Here, we describe the case of an 8-year-old boy diagnosed with severe haemophilia A shortly after birth.
Maria Sol Cruz +4 more
doaj +1 more source
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
New England Journal of Medicine, 2023 BACKGROUND Valoctocogene roxaparvovec delivers a B-domain-deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A.
J. Mahlangu +28 more
semanticscholar +1 more source
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
New England Journal of Medicine, 2022 BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
M. Ozelo +22 more
semanticscholar +1 more source
罕见病研究, 2022 Hemophilia is a group of hereditary hemorrhagic diseases. Hemorrhages mostly occur in musculoskeletal system and is the main cause for disability. Rehabilitation plays an important role in the comprehensive management in hemophilia.
Hemophilia Treatment Center Collaborative Network of China +1 more
doaj +1 more source
Gaceta Médica de México, 2023 Acquired hemophilia (AH) is an autoimmune hemostatic disorder mediated by autoantibodies directed against factor VIII: C. In 52% of cases, the cause is unknown or is not associated with other pathological entities; in the rest, there are concomitant factors: lupus, rheumatoid arthritis, cancer, pregnancy, and medications.
Jaime, García-Chávez +1 more
openaire +2 more sources
Science Advances, 2022 Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic ...
J. Han +18 more
semanticscholar +1 more source
WFH Guidelines for the Management of Hemophilia, 3rd edition
Haemophilia, 2020 Alok Srivastava 1 | Elena Santagostino 2 | Alison Dougall 3 | Steve Kitchen 4 | Megan Sutherland 5 | Steven W. Pipe 6 | Manuel Carcao 7 | Johnny Mahlangu 8 | Margaret V. Ragni 9 | Jerzy Windyga 10 | Adolfo Llinás 11 | Nicholas J. Goddard 12 | Richa Mohan
A. Srivastava +17 more
semanticscholar +1 more source
Hemophilia A with reduced coagulation factor Ⅺ: a case report and literature review
Zhongguo shuxue zazhi, 2023 Objective To investigate the possible molecular pathogenesis of a child with hemophilia A accompanied by coagulation factor Ⅺ reduction by testing coagulation-related indicators and genotyping in the child and his family.
Jie WANG +8 more
doaj +1 more source
Report on diagnosis and treatment of hemophilia in China 2023 [PDF]
Zhenduanxue lilun yu shijian, 2023 In recent years, China attaches great importance to the prevention and treatment of rare diseases. As one of the representative diseases of rare diseases, management of Hemophilia has made great progress in China.
XUE Feng, DAI Jing, CHEN Lixia, LIU Wei, ZHANG Houqiang, WU Runhui, SUN Jing, ZHANG Xinsheng, WU Jingsheng, ZHAO Yongqiang, WANG Xuefeng, YANG Renchi
doaj +1 more source
Research and Practice in Thrombosis and Haemostasis, 2022 The Hemophilia Joint Health Score (HJHS) was developed and validated to detect arthropathy in children. Additional evidence is required to show validity in adults.
J. St-Louis +21 more
semanticscholar +1 more source