Results 101 to 110 of about 49,324 (252)

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B [PDF]

, 2022
Pratima Chowdary, Susan Shapiro, Michael Makris, Gillian Evans, Sara Boyce, Kate Talks, Gerard Dolan, Ulrike M. Reiss, Mark Phillips, Anne Riddell, Maria R. Peralta, Michelle Quaye, David Patch, Edward G. D. Tuddenham, Allison Dane, Marie Watissée, Alison Long, Amit C. Nathwani   +17 more
openalex   +1 more source

A Comprehensive Approach: Addressing Psychological Needs in Gene Therapy for Haemophilia

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) represents a transformative development in the treatment of haemophilia, offering the potential for long‐lasting symptom control after a single administration. Although the scientific and medical aspects of this innovation have been widely studied, its psychological impact remains under‐addressed. Aims This paper
Aida Peyvandi, Ilaria Cutica, Isabella Garagiola, Flora Peyvandi   +3 more
wiley   +1 more source

Measurement of basal levels of factor VIIa in hemophilia A and B patients [see comments] [PDF]

, 1992
P Wildgoose, Yale Nemerson, L. L. Hansen, FE Nielsen, S Glazer, Ulla Hedner   +5 more
openalex   +1 more source

Monitoring of Nonacog Beta Pegol: One‐Stage Clotting Assay With Kaolin Reagent as a Practical Alternative to Chromogenic Methods

Haemophilia, EarlyView.
ABSTRACT Introduction Accurate monitoring of nonacog beta pegol (N9‐GP) is essential to ensure appropriate treatment and to avoid under‐ or overdosing, which may result in clinical complications. The product's extended half‐life, achieved through molecular modification, poses challenges for activity measurement, particularly when using a one‐stage ...
Marie Prudková, Petr Smejkal, Dagmar Chytrá, Jiřina Zavřelová, Gabriela Romanová, Miroslav Penka, Alena Buliková   +6 more
wiley   +1 more source

Evaluation of the Clinical Status of Patients with Inherited Bleeding Disorders in Diyala-Iraq

Diyala Journal of Medicine, 2019
Background: Inherited bleeding disorders are rare diseases that are both complicated and expensive to manage, they are classified into: coagulation factor disorders, vessel wall defect and platelet disorders.
Imad Ahmed Lateef
doaj   +2 more sources

Impact of mild to severe hemophilia on education and work by US men, women, and caregivers of children with hemophilia B: The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B‐HERO‐S) study [PDF]

, 2017
Susan Cutter, Don Molter, Spencer Dunn, Susan S. Hunter, Skye Peltier, Kimberly Haugstad, Neil Frick, Natalia Holot, David L. Cooper   +8 more
openalex   +1 more source

Quality of Life and Treatment Satisfaction in People With Haemophilia on Fitusiran Prophylaxis: Evidence From a Subset of ATLAS‐OLE Trial Participants Mainly From India

Haemophilia, EarlyView.
Alok Srivastava, Savita Rangarajan, Cecil Ross, Shariq Ali, Shauna Andersson, Laurel A. Menapace, Marja Puurunen, Marion Afonso   +7 more
wiley   +1 more source

Patient and Caregiver Preferences for Long‐Term Prophylactic Treatment of Bleeds in Haemophilia: A Discrete‐Choice Experiment

Haemophilia, EarlyView.
ABSTRACT Introduction The expansion of haemophilia treatment to include non‐factor prophylaxis provides new options with various benefits, risks, administration modes, and device types, necessitating trade‐offs in decision‐making. Aim This study evaluated prophylactic treatment preferences of people with haemophilia and their caregivers, assessing ...
Hui Lu, Travis Gould, Carmine Colavecchia, Joshua Coulter, Lisa J. Wilcox, Joseph C. Cappelleri, Stephanie A. Christopher, Alexis Sohn, Brett Hauber, Álvaro A. Gutiérrez‐Vargas, Nicola Dunn, Pratima Chowdary, Jennifer A. Whitty   +12 more
wiley   +1 more source

Engineering protein processing of the mammary gland to produce abundant hemophilia B therapy in milk [PDF]

, 2015
Jianguo Zhao, Weijie Xu, Jason W. Ross, Eric M. Walters, S. P. Butler, J. J. Whyte, Lindsey Kelso, Mostafa Fatemi, Nicholas C. Vanderslice, Keith Giroux, Lee D. Spate, Melissa Samuel, Cliff N. Murphy, Kevin D. Wells, Nick Masiello, Randall S. Prather, William H. Velander   +16 more
openalex   +1 more source

Supporting Patient‐Centred Decisions on Gene Therapy for Haemophilia A: A Conceptual Model

Haemophilia, EarlyView.
Giovanni Di Minno, Sarah Bigi, Serena Barello, Enrico Ferri Grazzi, Marco Follino, Maria Francesca Mansueto, Mariasanta Napolitano, Rita Carlotta Santoro, Giancarlo Castaman   +8 more
wiley   +1 more source