Zobrazeno 1 - 10
of 2 173
pro vyhledávání: '"Haemophilia B"'
Autor:
Sara Boyce, Simon Fletcher, April Jones, Ruchika Kohli, Sarah Mangles, Min Ong, Debra Pollard, Sujan Sivasubramaniyam, David Stephensen, Nicola Stoner, Rashid Kazmi
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-6 (2023)
Abstract With the first gene therapies for haemophilia approved by the European Commission, the US Food and Drug Administration, and the Medicines and Healthcare products Regulatory Agency, it is important to consider the remaining unmet needs and ch
Externí odkaz:
https://doaj.org/article/0ecd70c461494234bb755591f5067651
Publikováno v:
Hematology, Vol 28, Iss 1 (2023)
ABSTRACTStandard FIX prophylaxis for PWHB require frequent injections, which has led to the development of extended half-life products like rIX-FP (albutrepenonacog alfa) that has shown good efficacy in clinical studies. This ambispective study aims
Externí odkaz:
https://doaj.org/article/665a1b2ad7d044f5965a3410638193cf
Publikováno v:
The Journal of Haemophilia Practice, Vol 9, Iss 1, Pp 147-155 (2023)
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 f
Externí odkaz:
https://doaj.org/article/f3e41cac378a4959ab5aaae441a68bce
Autor:
Kwan Celia, Chan Anthony KC, Koh Pei Lin, Kadir Nur Insyirah Abdul, Decker Kay, Lim Chiew Ying, Lam Joyce Ching Mei
Publikováno v:
The Journal of Haemophilia Practice, Vol 9, Iss 1, Pp 96-102 (2022)
In children with haemophilia (CwH), central venous access devices (CVADs) are frequently placed to aid in the delivery of factor concentrates. In those who develop inhibitors, CVADs also allow for easy venous access and facilitation of immune toleran
Externí odkaz:
https://doaj.org/article/37e45183098049568464e187aec09045
Autor:
Idaira Rodriguez-Santana, Pronabesh DasMahapatra, Tom Burke, Zalmai Hakimi, José Bartelt-Hofer, Jameel Nazir, Jamie O’Hara
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-10 (2022)
Abstract Background The lifelong nature of haemophilia makes patient-centred and societal assessments of its impact important to clinical and policy decisions. Quantifying the humanistic and economic burden by severity is key to assessing the impact
Externí odkaz:
https://doaj.org/article/d130c614d9b748baa88ecef0ddcffef1
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-9 (2022)
Abstract Background Gene therapy has the potential to change the life experience of people with haemophilia and family members. Few studies have sought to explore the impact of gene therapy on both individuals and families. The aim of this study was
Externí odkaz:
https://doaj.org/article/12e45b570ed64589b8d8caada9e17e75
Autor:
Idaira Rodriguez-Santana, Pronabesh DasMahapatra, Tom Burke, Zalmai Hakimi, José Bartelt-Hofer, Jameel Nazir, Jamie O’Hara
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-9 (2022)
Abstract Background Haemophilia bears substantial humanistic and economic burden on children and their caregivers. Characterising the differential impact of severe versus moderate paediatric haemophilia is important for clinical and health policy dec
Externí odkaz:
https://doaj.org/article/ecfe8b8906354192bcc6037fa438dd51
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-9 (2021)
Abstract Background Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to bette
Externí odkaz:
https://doaj.org/article/c9837c198b5b422db946b102738f3dae
Autor:
Chaplin Steve, Birkedal Maj Friberg, Crilly Erica, Fletcher Simon, Garcia Sara, Mulders Greta, Myrin-Westesson Linda, Pollard Debra, Sanigorska Anna, Uitslager Nanda
Publikováno v:
The Journal of Haemophilia Practice, Vol 8, Iss 1, Pp 119-127 (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. Current clinical practice reduces the risk of bleeds, making differences difficult t
Externí odkaz:
https://doaj.org/article/215b834d74f04b0bbf71840e97f4f2b4
Publikováno v:
Journal of Biomedical Science, Vol 28, Iss 1, Pp 1-13 (2021)
Abstract Mainstay haemophilia treatment, namely intravenous factor replacement, poses several clinical challenges including frequent injections due to the short half-life of recombinant factors, intravenous administration (which is particularly chall
Externí odkaz:
https://doaj.org/article/5e718dda607841c981c6a76f2e806556