Results 31 to 40 of about 13,086,034 (342)
The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B
Blood Advances, 2018 : Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
J. Michael Soucie +4 more
doaj +1 more source
MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA
Mediterranean Journal of Hematology and Infectious Diseases, 2020 The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino +2 more
doaj +1 more source
Haematologica, 2020 The incidence of factor IX (FIX) inhibitors in severe hemophilia B (SHB) is not well defined. Frequencies of 3-5% have been reported but most studies to date have been small, including patients with different severities, and without prospective follow up
C. Male +13 more
semanticscholar +1 more source
A Case of Hemophilia B with Abdominal and Pelvic Pseudotumor
罕见病研究, 2022 Hemophilia B is a genetic disorder caused by coagulation factor Ⅸ(FⅨ) deficiency, mainly manifesting as joint, muscle and deep tissue bleeding. Hemophilia pseudotumor is a mass formed by soft tissue liquefaction and necrosis caused by repeated bleeding ...
ZHANG Jing +7 more
doaj +1 more source
Perinatal Gene Transfer to the Liver [PDF]
, 2011 The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM +6 more
core +1 more source
Cochlear implantation in hemophilia B—a rare case report
The Egyptian Journal of Otolaryngology, 2022 Background Hemophilia B is an X-linked inherited disease, mainly caused by deficiency of factor IX. Severity of the disease is manifested by the factor IX deficiency in the blood.
Chetan Yadav +3 more
doaj +1 more source
A review of evidence about behavioural and psychological aspects of chronic joint pain among people with haemophilia [PDF]
, 2014 Joint pain related to haemophilia affects large numbers of people and has a significant impact on their quality of life. This article reviews evidence about behavioural and psychological aspects of joint pain in haemophilia, and considers that evidence ...
Acharya +75 more
core +2 more sources
Etranacogene dezaparvovec for hemophilia B gene therapy
Therapeutic Advances in Rare Disease, 2021 The treatment landscape for hemophilia has been rapidly changing with introduction of novel therapies. Gene therapy for hemophilia is a promising therapeutic option for sustained endogenous factor production to mitigate the need for prophylactic ...
C. Thornburg
semanticscholar +1 more source
Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]
, 2001 Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A +5 more
core +1 more source
Molecular Therapy: Methods & Clinical Development, 2017 Gene therapy during neonatal and infant stages is a promising approach for hemophilia B, a congenital disorder caused by deficiency of blood coagulation factor IX (FIX).
Shunsuke Iizuka +4 more
doaj +1 more source