Haemophilia A and B are X-linked inherited bleeding disorders, resulting in the deficiency of clotting factor VIII and IX, respectively. Since the introduction of recombinant clotting factor concentrates in the early 1990s, the major safety concern for ...
Buckner Tyler W. +5 more
doaj +1 more source
Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors
Emicizumab is a bispecific monoclonal antibody that mimics factor VIII (FVIII) by binding to factors IXa and X to promote hemostasis in haemophilia A (HA) and HA with inhibitors (HA‐I). As emicizumab differs biochemically from FVIII, there is interest in
Patrick T. Ebbert +3 more
semanticscholar +1 more source
On a break with the X: the role of repair of double-stranded DNA breaks in X-linked disease [PDF]
The problem of managing free reactive DNA ends in eukaryotic cells has resulted in the development of a number of mechanisms in order to ensure that free ends are rendered non-reactive, or that the double-strand DNA breaks generating the free ends are ...
Cecceroni, Lucia +2 more
core +4 more sources
Inhibitor development in non-severe haemophilia across Europe [PDF]
Evidence about inhibitor formation in non-severe haemophilia and the potential role for clotting factor concentrate type is scant. It was the aim of this study to report inhibitor development in non-severe haemophilia patients enrolled in the European ...
Calizzani, Gabriele +13 more
core +3 more sources
Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5 (AAV5)‐mediated gene therapy approved for severe haemophilia A (HA).
E. Symington +9 more
semanticscholar +1 more source
''Sickle cell trait and haemophilia: a rare association''
In this paper we analyze the combination of HbAS disease and haemophilia A must be exceedingly rare. Because of this rarity we report the case of two brothers with sickle cell trait and major haemophilia A.
Hayat El Maataoui +2 more
doaj +1 more source
Acquired haemophilia A: a case report
Acquired haemophilia A is rare, autoimmune bleeding disorder caused by the production of autoantibodies against coagulation factor VIII (FVIII). The disorder affects 1.5 people per 1 million population a year, particularly the elderly.
Adam Stemplewski +3 more
doaj +1 more source
Developing a Two-Sided Decision Box to Facilitate Shared Decision-Making for Switching From Conventional to Pharmacokinetic-Tailored Prophylaxis in Haemophilia. [PDF]
Keepanasseril A +4 more
europepmc +3 more sources
Factor VIII products: key aspects of development, clinical research and use (part 2)
Continuous replacement therapy with clotting factor products can lead to serious complications in haemophilia A patients. One of potential reasons of such complications is an undesirable immune response to a blood clotting factor VIII (FVIII) product ...
Zh. I. Avdeeva +4 more
doaj +1 more source
PTH6: PATIENTS' PERCEPTIONS OF THE OUTCOMES OF TREATMENT WITH TOPICAL NSAIDS, RUBIFACIANTS AND ORAL PAIN REMEDIES. A PROSPECTIVE STUDY OF MEDICINES AVAILABLE THROUGH COMMUNITY PHARMACIES [PDF]
Obesity is a major health concern not only in the general population but also in patients with haemophilia. Little is known about the consequences of obesity for haemophilia patients.
Biere-Rafi, S. +5 more
core +1 more source

