Results 71 to 80 of about 21,171,014 (388)

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

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

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, EarlyView.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source

Living, Caring, Learning – The power of qualitative research in bleeding disorders care: One voice, from soloist to choir

The Journal of Haemophilia Practice
With over three decades of nursing experience, Simon reflects on his experience as a research nurse at the Oxford Haemophilia and Thrombosis Centre at a time of huge change for haemophilia care and treatment.
Fletcher Simon
doaj   +1 more source

Preventing perioperative bleeding in patients with inherited bleeding disorders [PDF]

, 2017
Data sources: Cochrane Cystic Fibrosis and Genetic Disorders Group's Coagulopathies Trials Register, a regularly updated database informed by trials identified within electronic databases including MEDLINE.
Beacher, Nicholas, Watterson, Colin
core   +1 more source

Laboratory measurement of factor replacement therapies in the treatment of congenital haemophilia: A United Kingdom Haemophilia Centre Doctors’ Organisation guideline

Haemophilia, 2019
Assay discrepancies can occur with laboratory monitoring of FVIII and FIX replacement therapy, particularly for the extended half‐life products. This guideline collates current published data and provides advice on appropriate choice of assays for ...
E. Gray, S. Kitchen, A. Bowyer, P. Chowdary, P. Jenkins, P. Murphy, S. Platton, A. Riddell, W. Lester   +8 more
semanticscholar   +1 more source

Traceless Regulation of Genetic Circuitry

Advanced Science, EarlyView.
Energy‐based, as opposed to molecular, control offers unprecedented improvements in key circuit parameters. This review summarizes the fundamentals of such traceless switches, categorizes them by trigger modalities, and compares and contrasts distinct advantages as well as shortcomings of each kind.
Gokberk Unal, Martin Fussenegger
wiley   +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

The effect of resistance exercise on strength and safety outcome for people with haemophilia: A systematic review

Haemophilia, 2020
Haemophilia is a congenital bleeding disorder with severe musculoskeletal complications. Resistance exercise is important to increase joint stability and to improve physical performance.
Barbara Wagner, S. Krüger, T. Hilberg, C. Ay, T. Hasenoehrl, Dominikus Huber, R. Crevenna   +6 more
semanticscholar   +1 more source

Construction of pathogenic Sec16a mutation mouse model using CRISPR/Cas9

Animal Models and Experimental Medicine, EarlyView.
Yaqiang Hu et al. engineered a pathogenic Sec16a mutant mouse model using CRISPR/Cas9 technology. They observed that the Sec16a mutant mice displayed diminished learning and memory capabilities, along with a limb‐clasping phenotype upon tail suspension.
Yaqiang Hu, Zhiyang Zeng, Xinyu Ming, Shuming Yin, Yuting Guan, Liangcai Gao, Dali Li   +6 more
wiley   +1 more source