Results 111 to 120 of about 13,086,034 (342)

Real-World Analysis of Dispensed IUs of Coagulation Factor IX and Resultant Expenditures in Hemophilia B Patients Receiving Standard Half-Life Versus Extended Half-Life Products and Those Switching from Standard Half-Life to Extended Half-Life Products

Journal of Managed Care & Specialty Pharmacy, 2018
BACKGROUND: Hemophilia B requires replacement therapy with factor IX (FIX) coagulation products to treat and prevent bleeding episodes. A recently introduced extended half-life (EHL) recombinant FIX replacement product provided the opportunity to compare
B. Tortella, J. Alvir, Margaret V. McDonald, D. Spurden, P. Fogarty, A. Chhabra, A. Pleil   +6 more
semanticscholar   +1 more source

Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system

Stem cell research & therapeutics, 2018
BackgroundReplacement therapy for hemophilia remains a lifelong treatment. Only gene therapy can cure hemophilia at a fundamental level. The clustered regularly interspaced short palindromic repeats–CRISPR associated nuclease 9 (CRISPR-Cas9) system is a ...
C. Lyu, Jun Shen, Rui Wang, Haihui Gu, Jian-Ping Zhang, F. Xue, Xiaofan Liu, Wei Liu, R. Fu, Liyan Zhang, Huiyuan Li, Xiaobing Zhang, Tao Cheng, Renchi Yang, Lei Zhang   +14 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

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

European Principles of Nursing Care for Persons With Inherited Bleeding Disorders

Haemophilia, EarlyView.
ABSTRACT Introduction A nurse is one of the core members of the multidisciplinary haemophilia team. However, what people with inherited bleeding disorders (PwBD) can expect from them has not yet been formally defined. Aim This project, initiated by the EAHAD Nurses Committee, aimed to establish Principles of Nursing Care (PoNC) for PwBD to promote high‐
Nanda Uitslager, Mary Kavanagh, Maj Birkedal, Linda Myrin‐Westesson, the EAHAD Nurses Committee   +4 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  

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

Navigating the Transition From Adolescence to Adulthood Among Young People With Severe Haemophilia: The Qualitative Phase of the TRANSHEMO Project

Haemophilia, EarlyView.
ABSTRACT Introduction Haemophilia causes spontaneous or prolonged bleeding due to a deficiency in clotting factor VIII (haemophilia A) or IX (haemophilia B). Although substitutive therapies and regular follow‐up can prevent severe haemorrhagic events, adherence to treatment remains a challenge.
Marie‐Anaïs Roques, Natacha Rosso‐Delsemme, Amandine Celli, Ngoc Anh Thu Nguyen, Martin Postzich, Sabine Castet, Yoann Huguenin, Annie Harroche, Anne Lienhart, Sandrine Meunier, Christine Biron‐Andréani, Florence Rousseau, Roseline d'Oiron, Yohann Repessé, Clémence Tabélé, Any Beltran Anzola, Thomas Sannié, Nicolas Giraud, Pascal Auquier, Hervé Chambost, Noémie Resseguier, The TRANSHEMO Study Group, Thémis Apostolidis, Laurent Ardillon, Karine Baumstarck, Sophie Bayart, Claire Berger, Marie‐Anne Bertrand, Carol Betsch, Annie Borel‐Derlon, Mohamed Boucekine, Thierry Calvez, Pierre Chamouni, Ségolène Claeyssens Donadel, Yannick Colle, Yesim Dargaud, Emmanuelle de Raucourt, Dominique Desprez, Céline Falaise, Alexandra Fournel, Birgit Frotscher, Valérie Gay, Fabienne Genre‐Volot, Jenny Goudemand, Yves Gruel, Benoît Guillet, Abel Hassoun, Audrey Hochart, Thierry Lambert, Aurélien Lebreton, Tanguy Leroy, Raphaël Marlu, Michèle Martin, Vanessa Milien, Fabrice Monpoux, Pierre Morange, Guillaume Mourey, Claude Negrier, Philippe Nguyen, Placide Nyombe, Caroline Oudot‐Challard, Brigitte Pan‐Petesch, Benoît Polack, Anne Rafowicz, Antoine Rauch, Delphine Rivaud, Pierre‐Simon Rohrlich, Pascale Schneider, Alexandra Spiegel, Cécile Stoven, Sophie Susen, Brigitte Tardy, Marc Trossaërt, Jean‐Baptiste Valentin, Stéphane Vanderbecken, Marie Viprey, Romain Voltzenlogel, Annelise Voyer‐Ebrard, Bénédicte Wibaut   +78 more
wiley   +1 more source

Physiological Aspects of Genetics [PDF]

, 1954
A considerable amount of evidence indicates that desoxyribonucleic acid is capable of duplicating itself, a property also possessed by genes. (By a self-duplicating material, we mean one which plays some essential role in its own production.) Watson ...
Horowitz, N. H., Owen, Ray D.
core   +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