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Postgraduate Medical Journal, 1997 Summary Although the nature of haemophilia has been understood for thousands of years, knowledge of its molecular genetics is recent. These X-linked bleeding disorders have diverse underlying DNA defects and, in 1992, DNA inversion within the X chromosome was found to explain half of the most serious cases of haemophilia A. The life-span
Cahill, Mary R., Colvin, B. T.
openaire +5 more sources
Haemophilia, 2021 Valoctocogene roxaparvovec is an investigational AAV5‐based factor VIII (FVIII) gene therapy that has demonstrated sustained clinical benefit in people with severe haemophilia A.
K. J. Pasi +11 more
semanticscholar +1 more source
Low immunogenicity of emicizumab in persons with haemophilia A
Haemophilia, 2021 Emicizumab is a humanised, bispecific monoclonal antibody mimicking the cofactor function of activated factor (F)VIII. It is indicated for routine prophylaxis of bleeding episodes in persons with haemophilia A (PwHA) with/without FVIII inhibitors.
C. Schmitt +10 more
semanticscholar +1 more source
Haemophilia, 2021 Severe haemophilia A (HA) has a major impact on health‐related quality of life (HRQoL).
M. Skinner +15 more
semanticscholar +1 more source
Gene Therapy in Hemophilia: A Transformational Patient Experience
Journal of Patient Experience, 2023 Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute ...
Enayet Rasul +7 more
doaj +1 more source
South African Family Practice, 2022 Background: Haemophilia A and B are X-linked recessive bleeding disorders resulting from a deficiency of factors VIII and IX, respectively. Early diagnosis and a comprehensive approach to management is mandatory.
Lethukuthula Mafisa +2 more
doaj +1 more source
Haemophilia, 2021 Few studies have examined the real‐world impact of haemophilia on daily activities and work productivity in people with severe haemophilia A (PWSHA).
J. O’Hara +7 more
semanticscholar +1 more source
The B-team: Equal but different?
The Journal of Haemophilia Practice, 2021 As a person with haemophilia B, I have known there are differences between haemophilia A and haemophilia B and their respective treatment throughout my life – though I was shocked when I learnt about the impact inhibitors can have when it comes to ...
Pembroke Luke
doaj +1 more source
Haemophilia specialist nurses’ perceptions of haemophilia B
The Journal of Haemophilia Practice, 2021 Some clinicians believe that haemophilia B is associated with less bleeding than haemophilia A, yet there appears to be little difference in health-related outcomes.
Chaplin Steve +9 more
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