Results 131 to 140 of about 22,643,381 (355)

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

Comparison of Real-World Dose and Consumption for Two Extended Half-Life Recombinant Factor VIII Products for the Treatment of Hemophilia A in the United States

Journal of Blood Medicine, 2022
Yanyu Wu,1 Shawn X Sun,1 Tao Fan2 1Global Evidence & Outcomes, Data Sciences Institute, Research and Development, Takeda Development Center Americas, Inc, Cambridge, MA, USA; 2US Health Economics and Outcomes Research, Takeda Pharmaceuticals USA, Inc ...
Wu Y, Sun SX, Fan T
doaj  

Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors.

Blood Advances, 2017
Emicizumab (ACE910), a recombinant humanized bispecific monoclonal antibody, provides factor VIII (FVIII) cofactor bridging function to restore hemostasis in people with hemophilia A.
M. Shima, H. Hanabusa, M. Taki, T. Matsushita, Tetsuji Sato, K. Fukutake, R. Kasai, K. Yoneyama, Hiroki Yoshida, K. Nogami   +9 more
semanticscholar   +1 more source

Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study

European Journal of Haematology, EarlyView.
ABSTRACT Objective To report data from an ITI chart review study (NCT03951103) for first‐time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A. Methods Retrospective and prospective real‐world data are reported from a non‐interventional, multicentre study of patients who had been or were currently ...
Robert Klamroth, Mahasen Al Saleh, Heidi Glosli, Michele Schiavulli, Benoît Guillet, Linda Bystrická, Anton Schönstein, Stefan Lethagen   +7 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  

Prominent Mutation of Intron 22 Inversion in Sporadic Hemophilia: Is It Worth the Antenatal Screening?

The Application of Clinical Genetics, 2022
Werasak Sasanakul, Ampaiwan Chuansumrit, Nongnuch Sirachainan, Praguywan Kadegasem Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, ThailandCorrespondence: Ampaiwan Chuansumrit, Department of Pediatrics ...
Sasanakul W, Chuansumrit A, Sirachainan N, Kadegasem P   +3 more
doaj  

Symposium: What is Hemophilia?: Introduction [PDF]

, 1954
William Dameshek
openalex   +1 more source

Successful use of emicizumab in a patient with refractory acquired hemophilia A and acute coronary syndrome requiring percutaneous coronary intervention

Research and Practice in Thrombosis and Haemostasis, 2019
Essentials Acquired hemophilia A is a rare bleeding disorder often accompanied by other comorbidities. We describe emicizumab use in acquired hemophilia A complicated by acute coronary syndrome.
Kathryn E. Dane, J. Lindsley, M. Streiff, A. Moliterno, Mian K. Khalid, S. Shanbhag   +5 more
semanticscholar   +1 more source

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF‐McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.
Mark W. Skinner, Manuela Albisetti, Jesús Ardila, Jan Astermark, Jan Blatny, Manuel Carcao, Pratima Chowdary, Nathan T. Connell, Miguel Crato, Yesim Dargaud, Roseline d'Oiron, Amy L. Dunn, Miguel A. Escobar, Carmen Escuriola‐Ettingshausen, Emna Gouider, Annie Harroche, Cedric Hermans, Victor Jimenez‐Yuste, Radoslaw Kaczmarek, Gili Kenet, Liane Khoo, Robert Klamroth, Florian Langer, David Lillicrap, Johnny Mahlangu, Christoph Male, Tadashi Matsushita, Sandrine Meunier, Wolfgang Miesbach, Beatrice Nolan, Johannes Oldenburg, Brian O'Mahony, Margareth Ozelo, Glenn F. Pierce, Gloria Ramos, Michael Recht, Olivia Romero‐Lux, Dawn Rotellini, Rita C. Santoro, Tammuella C. Singleton, Alok Srivastava, Sophie Susen, Kate Talks, Huyen Tran, Leonard A. Valentino, Jerzy Windyga, Renchi Yang, Maria Elisa Mancuso, also on behalf of AICE, AHAD‐AP, AHCDC, ATHN, CLAHT, EHC, French CRH & MHEMO, HTCCNC, NBDF, PEDNET, UKHCDO, WFH   +59 more
wiley   +1 more source

State of Iowa HIV and AIDS Surveillance Report, December 2004 [PDF]

, 2004
After taking a dip in 2003, HIV diagnoses were back up in 2004. There were 103 persons diagnosed in 2004, very close to our ten-year average of 100 cases per year. In 2003, there were 91 diagnoses.

core