Results 81 to 90 of about 50,045 (313)

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

Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

Journal of Translational Medicine, 2017
Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia.
David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava, Roland W. Herzog   +8 more
doaj   +1 more source

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

Health‐Related Quality of Life in Adult Patients With von Willebrand Disease From Germany: Results of the WIL‐QoL Study

Haemophilia, EarlyView.
ABSTRACT Introduction Assessment of health‐related quality of life (HRQoL) is relatively new in von Willebrand disease (VWD). So far, generic questionnaires have mainly been used for HRQoL assessment in VWD. Aims To assess generic and disease‐specific HRQoL in adult VWD patients and compare HRQoL with the general German population.
Sylvia von Mackensen, Carolin Moorthi, Ronald Fischer, Susan Halimeh, Christine Heller, Wolfgang Miesbach, Freimut H. Schilling, Cornelia Wermes, Guenter Auerswald, the WIL‐QoL study group, Karin Beutel, Georg Goldmann, Bettina‐Kemkes Matthes, Robert Klamroth, Ralf Knöfler, Christoph Male, Johannes Oldenburg, Ute Scholz   +17 more
wiley   +1 more source

Mild-severe hemophilia B impacts relationships of US adults and children with hemophilia B and their families: results from the B-HERO-S study

Patient Related Outcome Measures, 2019
Susan Cutter,1 Christine Guelcher,2 Susan Hunter,3 Dawn Rotellini,4 Spencer Dunn,5 David L Cooper61Penn Comprehensive Hemophilia and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA, USA; 2Comprehensive Hemostasis and ...
Cutter S, Guelcher C, Hunter S, Rotellini D, Dunn S, Cooper DL   +5 more
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

LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]

, 2010
Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA, Coutelle, C, David, A, Harbottle, R, Themis, M, Waddington, SN   +5 more
core   +3 more sources

Safety and Effectiveness of Emicizumab in People With Haemophilia A Enrolled in the ATHN 7 Haemophilia Natural History Study

Haemophilia, EarlyView.
ABSTRACT Background The Natural History Study of the Safety, Effectiveness, and Practice of Treatment for People with Haemophilia (ATHN 7) monitors use of contemporary haemophilia therapies, including emicizumab, a bispecific antibody substituting for activated factor (F)VIII in people with haemophilia A (HA).
Tyler W. Buckner, Shannon L. Carpenter, Nabil Daoud, Christine L. Kempton, Lucy Lee, Lynn Malec, Thomas W. McLean, Paul Morton, Carrie O'Neill, Janice M. Staber, Michael Wang, Stacy E. Croteau, Michael Recht   +12 more
wiley   +1 more source

Cost Analysis of Radioactive Versus Arthroscopic Synovectomy in Haemophilia: A Brazilian Modelling Approach

Haemophilia, EarlyView.
ABSTRACT Introduction Radioactive synovectomy (RSO) is an alternative to arthroscopic synovectomy (AR), for treating synovitis. RSO reduces the number of hemarthroses, being less invasive and less costly. Objective To analyse the costs of RSO compared to AR in the treatment of patients with haemophilia in Brazil, using real‐world data (RSO) and ...
Sylvia Thomas, Mariana Battazza, Vinicius Bednarczuk de Oliveira, Indianara Galhardo, Sergio Augusto Lopes de Souza   +4 more
wiley   +1 more source

CO27 | Intracerebral hemorrhage in hemophilia A and B: a 10-year prospective multicenter study in Italy (2012–2022)

Bleeding, Thrombosis and Vascular Biology
Background and Aims: Intracerebral hemorrhage (ICH) is a severe and life-threatening complication in patients with hemophilia, often resulting in high mortality and long-term neurological impairment.
doaj   +1 more source