Results 71 to 80 of about 49,324 (252)

Children and Adolescents With Localised Non‐Rhabdomyosarcoma Soft Tissue Sarcoma: Results of the CWS‐96 and CWS‐2002P Prospective Trials With Reclassification of the Trial Data Incorporating the Recent Soft Tissue Sarcoma Registry

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Here we report the results of the first comprehensive European trials for all soft tissue sarcoma entities, which aimed to develop a new risk stratification system, limit radiotherapy and chemotherapy for low‐/standard‐risk patients, and evaluate different chemotherapy regimens for high‐risk patients.
Amadeus T. Heinz, Anton Schönstein, Ewa Koscielniak, Martin Ebinger, Jörg Fuchs, Semi Harrabi, Christian Vokuhl, Roland Imle, Sabine Stegmaier, Ines B. Brecht, Gustaf Ljungman, Hanna Juntti, Ruth Ladenstein, Bernarda Kazanowska, Thomas Klingebiel, Kristian W. Pajtler, Monika Sparber‐Sauer   +16 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

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

Cyclosporine treatment of acquired hemophilia due to factor VIII antibodies [PDF]

, 2000
Acquired hemophilia, caused by autoantibodies against coagulation factor VIII, is usually treated with steroids, cyclophosphamide, intravenous gammaglobulins and sporadically other drugs.
Baele, Gaston, Derom, Eric, Petrovic, Mirko   +2 more
core  

Randomized trial of a DVD intervention to improve readiness to self-manage joint pain [PDF]

, 2011
Report of a randomized controlled trial of a motivational intervention to promote self-management of joint painA DVD (digital video disk) intervention to increase readiness to self-manage joint pain secondary to hemophilia was informed by a 2-phase ...
Abraham, Altman, Angst, Barlow, Barlow, Barry, Biller, Bode, Carr, Choiniere, Dijkstra, Elander, Elander, Elander, Fischer, Glenn, Habib, Hadjistavropoulos, Hays, Hays, Heapy, Jensen, Jensen, Jensen, Jensen, Kazis, Kerns, Kerns, Kerns, LeFort, Liao, Luck, McCracken, Moore, Nielsen, Prochaska, Richmond, Ruta, Szende, Turk, Turk, Von Korff, Vowles, Warsi   +43 more
core   +2 more sources

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

Small Methods, EarlyView.
The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J. Mitchell, Xuexiang Han   +6 more
wiley   +1 more source

Real‐World Effectiveness and Safety of Damoctocog Alfa Pegol in Severe and Nonsevere Patients With Hemophilia A From the Prospective, Multinational, Ongoing HEM‐POWR Study

European Journal of Haematology, EarlyView.
ABSTRACT Objectives To assess the effectiveness and safety of damoctocog alfa pegol in patients with severe and nonsevere hemophilia A in the fifth interim analysis of the ongoing HEM‐POWR study. Methods HEM‐POWR (NCT03932201) is a multinational, Phase 4, prospective observational study.
Mark T. Reding, María Teresa Alvarez Román, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg   +7 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  

Missense mutations near the N-glycosylation site of the A2 domain lead to various intracellular trafficking defects in coagulation factor VIII [PDF]

, 2017
Citation: Wei, W., Zheng, C. L., Zhu, M., Zhu, X. F., Yang, R. C., Misra, S., & Zhang, B. (2017). Missense mutations near the N-glycosylation site of the A2 domain lead to various intracellular trafficking defects in coagulation factor VIII.
Misra, Saurav, Wei, W., Yang, R. C., Zhang, B., Zheng, C. L., Zhu, M., Zhu, X. F.   +6 more
core   +1 more source