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 …

The hemophilias—from royal genes to gene therapy

PM Mannucci, EGD Tuddenham - New England Journal of …, 2001 - Mass Medical Soc
Of the various types of hemophilia, the most common of these lifelong bleeding disorders
are due to an inherited deficiency of factor VIII or factor IX (Table 1). The genes for these …

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 …

[PDF][PDF] Unequal, Unfair, Ineffective and Inefficient Gender Inequity in Health: Why it exists and how we can change it

G Sen, A George, P Ostlin, S Ramos - 2007 - repositorio.cedes.org
Gender inequality damages the health of millions of girls and women across the globe. It can
also be damaging to men's health despite the many tangible benefits it gives men through …

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

CS Manno, AJ Chew, S Hutchison… - Blood, The Journal …, 2003 - ashpublications.org
Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation
factor IX (F. IX). Previously, we established an experimental basis for gene transfer as a …

Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

JS Powell, NC Josephson, D Quon… - Blood, The Journal …, 2012 - ashpublications.org
Current factor VIII (FVIII) products display a half-life (t1/2) of∼ 8-12 hours, requiring frequent
intravenous injections for prophylaxis and treatment of patients with hemophilia A. rFVIIIFc is …

Impact of inhibitors on hemophilia a mortality in the U nited S tates

CE Walsh, JM Soucie, CH Miller… - American journal of …, 2015 - Wiley Online Library
The previously published mortality studies are limited in hemophilia populations but suggest
that there is no increased risk of mortality in factor VIII inhibitor patients. This retrospective …

European principles of haemophilia care

BT Colvin, J Astermark, K Fischer, A Gringeri… - …, 2008 - Wiley Online Library
As the management of haemophilia is complex, it is essential that those with the disorder
should have ready access to a range of services provided by a multidisciplinary team of …

Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients

AD Shapiro, MV Ragni, LA Valentino… - Blood, The Journal …, 2012 - ashpublications.org
Current factor IX (FIX) products display a half-life (t1/2) of∼ 18 hours, requiring frequent
intravenous infusions for prophylaxis and treatment in patients with hemophilia B. This open …

Mortality in congenital hemophilia A–a systematic literature review

CRM Hay, F Nissen, SW Pipe - Journal of Thrombosis and …, 2021 - Wiley Online Library
Against a background of a rapidly evolving treatment landscape, a contemporary, evidence‐
based consolidated understanding of mortality in people with congenital hemophilia A …