Results 71 to 80 of about 50,045 (313)

Pharmacokinetic Study of Recombinant Human Factor IX in Previously Treated Patients with Hemophilia B in Taiwan

Journal of the Formosan Medical Association, 2007
The pharmacokinetic (PK) study of recombinant human factor IX (rFIX) has been done in patients with hemophilia B in areas other than Taiwan. However, wide patient-related variability in recovery is noted and there is no PK study of rFIX in Taiwanese ...
Hsiu-Hao Chang, Yung-Li Yang, Mei-Hua Hung, Woei Tsay, Ming-Ching Shen   +4 more
doaj   +1 more source

Anti-CD20 as the B-Cell Targeting Agent in a Combined Therapy to Modulate Anti-Factor VIII Immune Responses in Hemophilia a Inhibitor Mice [PDF]

, 2014
Neutralizing antibody formation against transgene products can represent a major complication following gene therapy with treatment of genetic diseases, such as hemophilia A.
Carol H. Miao, Chao Lien Liu, Chérie L. Butts, Jacqueline Lin, Peiqing Ye   +4 more
core   +1 more source

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, EarlyView.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

A viable mouse model of factor X deficiency provides evidence for maternal transfer of factor X. [PDF]

, 2008
BackgroundActivated factor X (FXa) is a vitamin K-dependent serine protease that plays a pivotal role in blood coagulation by converting prothrombin to thrombin. There are no reports of humans with complete deficiency of FX, and knockout of murine F10 is
Arruda, VR, Chu, K, Golden, JA, Herzog, RW, High, KA, Labosky, PA, Margaritis, P, Tai, SJ   +7 more
core   +2 more sources

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B [PDF]

, 2022
Pratima Chowdary, Susan Shapiro, Michael Makris, Gillian Evans, Sara Boyce, Kate Talks, Gerard Dolan, Ulrike M. Reiss, Mark Phillips, Anne Riddell, Maria R. Peralta, Michelle Quaye, David Patch, Edward G. D. Tuddenham, Allison Dane, Marie Watissée, Alison Long, Amit C. Nathwani   +17 more
openalex   +1 more source

Current gynaecological management of women and girls with bleeding disorders in the United Kingdom: A UKHCDO haemophilia treatment centre survey and evaluation of real‐world clinical practice for the British Journal of Haematology

British Journal of Haematology, EarlyView.
Summary Girls and women with bleeding disorders (GWBD) comprise more than half of all registered patients with bleeding disorders in the UK National Haemophilia Database. The gynaecological care of GWBD, until recently, has not been prioritised despite high health burdens, where four of every five patients experience heavy menstrual bleeding (HMB).
Laura Knox, Imogen Swart‐Rimmer, Naim Rahimi, Callum Harris, Lugain Abdalla, Gary Benson, Clare Brown, Helen Campbell, Ana Carvalhosa, Justin T. Clarke, Sarah Garside, Claire Lentaigne, Jayna Mistry, Priyanka Raheja, Cora Warren, Rezan Abdul‐Kadir, Gill Lowe, Nicola Curry   +17 more
wiley   +1 more source

Suboptimal screening and correction of maternal iron deficiency anaemia in the general population and in inherited bleeding disorders: A population‐based cohort study

British Journal of Haematology, EarlyView.
Summary Iron deficiency anaemia (IDA) is associated with adverse pregnancy outcomes globally. Women with inherited bleeding disorders are at increased risk, with scarce data on rates of IDA screening and correction during pregnancy. The impact of correction on outcomes is unclear.
Arafat Ul Alam, Padma Kaul, Venu Jain, Cynthia Wu, Haowei (Linda) Sun   +4 more
wiley   +1 more source

Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa‐PROTECT Main Study

European Journal of Haematology, EarlyView.
ABSTRACT Background In the earlier PROTECT VIII Kids study (NCT01775618), damoctocog alfa pegol was efficacious for prevention and treatment of bleeds in children aged < 12 years with severe haemophilia A. Objective Assess the safety of damoctocog alfa pegol, including hypersensitivity and loss of efficacy (LoE) due to an immune response to ...
Margareth C. Ozelo, Matteo Luciani, Heidi Glosli, Kaan Kavakli, Nasrin Samji, Gregory C. Makris, Claudia Tueckmantel, Monika Maas Enriquez, Luciana C. Oliveira, Shveta Gupta, Mario Guillermo Arbesú, Mauro Davoli, Anthony K. C. Chan, Maria E. Mancuso   +13 more
wiley   +1 more source

Spectrum of hemophilia in Diyala-Iraq

Diyala Journal of Medicine, 2019
Background: Deficiencies of coagulation factors have been recognized for centuries. The most common inherited factor deficiencies are the hemophilias, X-linked diseases caused by deficiency of factor VIII (hemophilia A) or factor IX ( hemophilia B ...
Imad Ahmed Lateef, Hayder Jassim Hamood, Hayder Jassim Hamood, Oday Abbood Khaleel   +3 more
doaj   +2 more sources

Patient and parent preferences for characteristics of prophylactic treatment in hemophilia

Patient Preference and Adherence, 2015
Roberto Furlan,1 Sangeeta Krishnan,2 Jeffrey Vietri3 1Advanced Methods, Kantar Health, Epsom, Surrey, UK; 2Global Health Economics and Outcomes Research, Biogen, MA, USA; 3Health Outcomes, Kantar Health, Milan, Italy Introduction: New longer-acting ...
Furlan R, Krishnan S, Vietri J
doaj