Results 61 to 70 of about 22,562,840 (346)

Haemophilia A management with emicizumab: A survey of haematologists in the United States

The Journal of Haemophilia Practice, 2022
Emicizumab is a bispecific monoclonal antibody approved in the United States (US) for the treatment of people with haemophilia A (PwHA) with or without factor VIII (FVIII) inhibitors.
Patel Anisha M., Owens Wendy E., Poulos Kathleen, Raimundo Karina, Wong Gabriel, Bernard Janet, Powers Janet, Ko Richard H.   +7 more
doaj   +1 more source

The effect of emicizumab prophylaxis on health‐related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study

Haemophilia, 2018
Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health‐related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis.
J. Oldenburg, J. Mahlangu, W. Buján, P. Trask, M. Callaghan, G. Young, E. Asikanius, F. Peyvandi, E. Santagostino, R. Kruse-Jarres, C. Négrier, C. Kessler, Jin Xu, J. Windyga, M. Shima, S. von Mackensen   +15 more
semanticscholar   +1 more source

Inhibitor development in haemophilia according to concentrate Four-year results from the European HAemophilia Safety Surveillance (EUHASS) project [PDF]

, 2015
Inhibitor development represents the most serious side effect of haemophilia treatment. Any difference in risk of inhibitor formation depending on the product used might be of clinical relevance.
Calizzani, G., Fischer, K., Gatt, A., Gilman, E., Iorio, A., Lambert, T., Lassila, R., Makris, M., Participants, EUHASS, Peyvandi, F., Windyga, J.   +10 more
core   +5 more sources

Recent Advances in mRNA Delivery Systems for Cancer Therapy

Advanced Science, EarlyView.
This review systematically investigates the applications of mRNA therapy in cancer treatment, with particular emphasis on nonviral delivery systems, targeting strategies, stimulus‐responsive systems, and local delivery methods. Concluding with a meticulous evaluation, the review sheds light on the prevailing challenges while illuminating promising ...
Zheng Zhang, Ya‐Nan Fan, Si‐Qi Jiang, Ya‐Jing Ma, Yao‐Ru Yu, Yu‐Xin Qing, Qian‐Ru Li, Yi‐Lin Liu, Song Shen, Jun Wang   +9 more
wiley   +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, Chan, JK, Howe, SJ, McKay, TR, Rahim, AA, Waddington, SN, Ward, NJ   +6 more
core   +1 more source

The UK Infected Blood Inquiry: A Personal Reflection. [PDF]

Haemophilia
Haemophilia, EarlyView.
Lee CA.
europepmc   +2 more sources

From the voices of people with haemophilia A and their caregivers: Challenges with current treatment, their impact on quality of life and desired improvements in future therapies

Haemophilia, 2019
Haemophilia A is a chronic disease requiring frequent intravenous infusions of recombinant factor VIII. Previous studies have shown that challenges associated with current treatments may have significant impacts on quality of life (QoL) that are as ...
R. E. Wiley, C. P. Khoury, A. Snihur, Marni Williams, D. Page, N. Graham, L. Laudenbach, Cindy Milne‐Wren, J. Stoffman   +8 more
semanticscholar   +1 more source

Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report

American Journal of Hematology, EarlyView.
ABSTRACT Hereditary hemorrhagic telangiectasia (HHT, Osler‐Weber‐Rendu disease) is the second most common inherited bleeding disorder worldwide, affecting approximately 1 in 5000 people. Development of disease‐modifying and efficacious hemostatic agents to treat HHT has finally begun after decades without such medical therapies.
Hanny Al‐Samkari, Raj S. Kasthuri, Hans‐Jurgen Mager, Jenny Y. Zhou, Marcelo M. Serra, Bethany T. Samuelson‐Bannow, Layla N. Van Doren, Jay F. Piccirillo, Marianne S. Clancy, Keith R. McCrae, Sonia M. Thomas, Antoni Riera‐Mestre, Allyson M. Pishko, Sarah Sewaralthahab, James R. Gossage, Vivek N. Iyer, Cedric Hermans, Adrienne Hammill, Ingrid Winship, Meir Mei‐Zahav, Annette von Drygalski, Scott Olitsky, Marie E. Faughnan   +22 more
wiley   +1 more source

Living, Caring, Learning - Changing mindsets and enabling goals for people with haemophilia

The Journal of Haemophilia Practice
In a nursing career spanning almost five decades, Anne Louise has spent over 30 years caring for people with haemophilia in South Africa. She reflects on the importance of prophylaxis and how tailoring treatment to meet individual needs not only helps to
Cruickshank Anne-Louise
doaj   +1 more source

Clinical profile of children with haemophilia at the University Hospital of Brazzaville

The Journal of Haemophilia Practice, 2020
Haemophilia is a rare hereditary haemorrhagic disease caused by coagulation factor VIII (haemophilia A) or IX (haemophilia B) deficiency. Very few data exist on this disease in Congo.
Galiba Atipo Tsiba FO, Ngolet OL, Ngakengni NY, Ollandzobo Ikobo LC, Nziengui-Mboumba JV, Elira Dokekias A   +5 more
doaj   +1 more source