Results 81 to 90 of about 12,413,959 (307)

New Technologies Empower Hemophilic Arthropathy Treatment: Focusing on Regenerative Medicine, Molecular Targeting, and Gene Therapy

Medicine Bulletin, EarlyView.
ABSTRACT Hemophilic arthropathy (HA), a major complication of hemophilia, is a chronic osteoarthropathy driven by repeated joint bleeding. Although traditional therapies such as coagulation factor replacement, physical rehabilitation, and surgery can manage symptoms, they cannot fundamentally repair established joint damage or break the pathological ...
Lin Sun, Yingnan Wang, Leyuan Zhao, Liqiang Lu, Fushan Hou, Chuan Yin, Kun Zhang, Yongqiang Hao, Jingke Fu   +8 more
wiley   +1 more source

Distribution and predictors of haemophilia-related costs in the United Kingdom: analysis of the CHESS I and CHESS II burden of illness studies

BMC Health Services Research
Background Few studies have evaluated direct medical or societal costs of haemophilia in the United Kingdom (UK), and how patient characteristics impact future costs is uncertain.
Ione Woollacott, Amit Chhabra, Tom Burke, Jack Brownrigg, Lucy Richardson, Enrico Ferri Grazzi, Jamie O’Hara, Josie Godfrey, Michael Laffan   +8 more
doaj   +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

Efficacy and safety of long‐acting recombinant fusion protein linking factor IX with albumin in haemophilia B patients undergoing surgery

Haemophilia, 2016
Recombinant factor IX fusion protein (rIX‐FP) has been developed to improve the pharmacokinetic (PK) profile of factor IX (FIX), allowing maintenance of desired FIX activity between injections at extended intervals, ultimately optimizing haemophilia B ...
Claude Negrier, F. Karim, Lynda Mae Lepatan, A. Lienhart, M. López-Fernández, Johnny Mahlangu, Ingrid Pabinger, Yanyan Li, D. Wolko, Christine Voigt, Iris Jacobs, E. Santagostino   +11 more
semanticscholar   +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

Simplifying surgery in haemophilia B: Low factor IX consumption and infrequent infusions in surgical procedures with rIX-FP [PDF]

, 2020
Julie Curtin, Elena Santagostino, Faraizah Abdul Karim, Yanyan Li, Wilfried Seifert, Claude Négrier   +5 more
openalex   +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

Lessons from a qualitative study of treatment experiences and perceptions in people with haemophilia in France

The Journal of Haemophilia Practice
Haemophilia A and B (HA/HB) are congenital, X-linked recessive bleeding disorders caused by deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively.
Castet Sabine-Marie, Sepot-Boucherit Lola, Beranger Nicolas, Delienne Stephanie, Chamouard Valérie   +4 more
doaj   +1 more source

The spectrum of bleeding in women and girls with haemophilia B

Haemophilia, 2018
Although hemophilia B affects 1 in 25,000 males there may be 3 female hemophilia B carriers per affected male. This clinical review highlights the unique challenges faced by hemophilia B carriers including the under‐recognition of bleeding symptoms ...
Janice M. Staber, S. Croteau, Joanna A. Davis, Eric F. Grabowski, P. Kouides, Robert F Sidonio   +5 more
semanticscholar   +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