Results 31 to 40 of about 22,597,991 (289)

Recombinant factorVIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A [PDF]

, 2015
This work was supported by funding from Biogen, including funding for the editorial and writing support in the the development of this ...
Allen, G, Barnes, C, Cristiano, LM, Gambino, G, Kulkarni, R, Liesner, R, Mahlangu, J, Neelakantan, S, Nolan, B, Pasi, J, Pierce, GF, Young, G   +11 more
core   +1 more source

Practices and challenges for hemophilia management under resource constraints in Thailand

Orphanet Journal of Rare Diseases, 2023
Hemophilia is an inherited bleeding disorder caused by deficiency of a specific coagulation factor. Factor VIII deficiency is responsible for hemophilia A while factor IX deficiency is responsible for hemophilia B. As per the 2020 annual global survey by
Chatphatai Moonla, Darintr Sosothikul, Bunchoo Pongtanakul, Bundarika Suwanawiboon, Chanchai Traivaree, Rungrote Natesirinilkul, Nongnuch Sirachainan, Pantep Angchaisuksiri   +7 more
doaj   +1 more source

Establishment and Evolution of China National Hemophilia Registry

罕见病研究, 2022
Hemophilia is an inherited bleeding disorder and a type of rare disease that is hereditary, lifelong and disabling. The establishment of a National Hemophilia Registry is foundational to treating hemophilia.
XUE Feng, YANG Renchi
doaj   +1 more source

Hemophilia A gene therapy: current and next-generation approaches

Expert Opinion on Biological Therapy, 2021
Introduction Hemophilia comprises a group of X-linked hemorrhagic disorders that result from a deficiency of coagulation factors. The disorder affects mainly males and leads to chronic pain, joint deformity, reduced mobility, and increased mortality ...
S. Pipe, Gil Gonen-Yaacovi, O. Segurado
semanticscholar   +1 more source

A review of evidence about behavioural and psychological aspects of chronic joint pain among people with haemophilia [PDF]

, 2014
Joint pain related to haemophilia affects large numbers of people and has a significant impact on their quality of life. This article reviews evidence about behavioural and psychological aspects of joint pain in haemophilia, and considers that evidence ...
Acharya, Barlow, Barry, Buhrman, Buzzard, Cour, Eckman, Elander, Elander, Elander, Elander, Elander, Ersek, Fischer, Fischer, Fishbain, Galen, Genderen, Gil, Hermans, Hilberg, Holstein, Humphries, Ingerslev, Jensen, Jensen, Karimi, Korff, Laforest, LeFort, Lorig, Lorig, Lorig, Lusher, Mackensen, McCracken, McCracken, McCracken, McCracken, Moore, Moore, Mulvany, Neufeld, Niscola, O'Mahony, Penica, Poon, Riley, Risdon, Rodriguez-Merchan, Royal, Santavirta, Santavirta, Schramm, Schulz, Sherry, Solomon, Solovieva, Spitzer, Stover, Swirsky-Sacchetti, Szende, Thorn, Turk, Turk, Varni, Varni, Varni, Varni, Wallny, Wilson, Witkop, Witkop, Wright, Zawilska, Zhou   +75 more
core   +2 more sources

Prophylaxis in Hemophilia

Clinical and Biomedical Research, 2023
Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich, Dayenne Catelli, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

Hemophilia: a biography on therapeutical approaches

Clinical and Biomedical Research, 2023
The history of hemophilia is ancient, with descriptions dated to the 2nd century AD. The first modern narratives appeared in 1800s, when total blood transfusion was the only available treatment and life expectancy was remarkably low.
Dayenne Catelli, Júlia Plentz Portich, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors

New England Journal of Medicine, 2018
BACKGROUND Emicizumab is a bispecific monoclonal antibody that bridges activated factor IX and factor X to replace the function of missing activated factor VIII, thereby restoring hemostasis.
J. Mahlangu, J. Oldenburg, I. Paz‐Priel, C. Négrier, M. Niggli, E. Mancuso, C. Schmitt, V. Jiménez‐Yuste, C. Kempton, C. Dhalluin, M. Callaghan, W. Buján, M. Shima, J. Adamkewicz, E. Asikanius, G. Levy, R. Kruse-Jarres   +16 more
semanticscholar   +1 more source

BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.

New England Journal of Medicine, 2020
BACKGROUND Factor VIII replacement products have improved the care of patients with hemophilia A, but the short half-life of these products affects the patients' quality of life. The half-life of recombinant factor VIII ranges from 15 to 19 hours because
B. Konkle, A. Shapiro, D. Quon, J. Staber, R. Kulkarni, M. Ragni, E. S. Chhabra, S. Poloskey, K. Rice, S. Katragadda, J. Fruebis, C. Benson   +11 more
semanticscholar   +1 more source

Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A. [PDF]

, 2012
nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Abriss, Daniela, Milanov, Peter, Pepperkok, Rainer, Quade-Lyssy, Patricia, Roth, Stefanie D., Schüttrumpf, Jörg, Seifried, Erhard, Simpson, Jeremy C., Tonn, Torsten, Ungerer, Christopher   +9 more
core   +2 more sources