Haemophilia
E Berntorp, K Fischer, DP Hart, ME Mancuso… - Nature reviews Disease …, 2021 - nature.com
Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or
deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease …
deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease …
Modern haemophilia care
E Berntorp, AD Shapiro - The Lancet, 2012 - thelancet.com
Haemophilia care has undergone substantial improvements during the past 40–50 years.
Early clotting factor concentrates were not sufficiently refined to enable self-administered …
Early clotting factor concentrates were not sufficiently refined to enable self-administered …
Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries
Background: The large observed variability in hemophilia prevalence prevents robust
estimation of burden of disease. Objective: To estimate the prevalence and prevalence at …
estimation of burden of disease. Objective: To estimate the prevalence and prevalence at …
Guidelines for the management of hemophilia
A Srivastava, AK Brewer, EP Mauser‐Bunschoten… - …, 2013 - Wiley Online Library
Hemophilia is a rare disorder that is complex to diagnose and to manage. These evidence‐
based guidelines offer practical recommendations on the diagnosis and general …
based guidelines offer practical recommendations on the diagnosis and general …
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model
T Kitazawa, T Igawa, Z Sampei, A Muto, T Kojima… - Nature medicine, 2012 - nature.com
Hemophilia A is a bleeding disorder resulting from coagulation factor VIII (FVIII) deficiency.
Exogenously provided FVIII effectively reduces bleeding complications in patients with …
Exogenously provided FVIII effectively reduces bleeding complications in patients with …
The world federation of hemophilia annual global survey 1999‐2018
JS Stonebraker, PHB Bolton‐Maggs, M Brooker… - …, 2020 - Wiley Online Library
Abstract Introduction The World Federation of Hemophilia (WFH) strives to achieve care for
all patients with inherited bleeding disorders through research, advocacy, capacity building …
all patients with inherited bleeding disorders through research, advocacy, capacity building …
Pathophysiology of hemophilic arthropathy and potential targets for therapy
AE Pulles, SC Mastbergen, REG Schutgens… - Pharmacological …, 2017 - Elsevier
Hemophilia is a congenital clotting factor deficiency characterized by spontaneous and
trauma-related bleeding. Spontaneous bleeding shows a predilection for joints, and …
trauma-related bleeding. Spontaneous bleeding shows a predilection for joints, and …
Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A
T Calvez, H Chambost… - Blood, The Journal …, 2014 - ashpublications.org
Six recombinant factor VIII (rFVIII) products have been marketed worldwide. In 2013, the
Research of Determinants of Inhibitor Development (RODIN) study group reported an …
Research of Determinants of Inhibitor Development (RODIN) study group reported an …
[HTML][HTML] A molecular revolution in the treatment of hemophilia
JSS Butterfield, KM Hege, RW Herzog, R Kaczmarek - Molecular Therapy, 2020 - cell.com
For decades, the monogenetic bleeding disorders hemophilia A and B (coagulation factor
VIII and IX deficiency) have been treated with systemic protein replacement therapy. Now …
VIII and IX deficiency) have been treated with systemic protein replacement therapy. Now …
[HTML][HTML] Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII
H Chen, M Shi, A Gilam, Q Zheng, Y Zhang… - Scientific reports, 2019 - nature.com
Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency
caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as …
caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as …