CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice [PDF]
Scientific Reports, 2017 Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology.
Tsukasa Ohmori +10 more
doaj +2 more sources
Perioperative management of a patient with haemophilia B and PSVT undergoing radiofrequency ablation: A case report [PDF]
Journal of International Medical Research, 2023 Haemophilia B is a rare inherited bleeding disorder in which patients have impaired coagulation. This study describes a patient with Haemophilia B and paroxysmal supraventricular tachycardia (PSVT) who underwent radio frequency catheter ablation (RFCA ...
Yu Gan, Liping Ma, Pihua Fang
doaj +2 more sources
Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe [PDF]
Orphanet Journal of Rare Diseases, 2021 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.
Tom Burke +5 more
doaj +2 more sources
Once‐weekly prophylactic treatment vs. on‐demand treatment with nonacog alfa in patients with moderately severe to severe haemophilia B [PDF]
Haemophilia, 2016 Limited data are available on optimal prophylaxis regimens of factor IX (FIX) replacements for patients with haemophilia B.
K. Kavaklı +8 more
semanticscholar +2 more sources
Optimal trough levels in haemophilia B: Raising expectations. [PDF]
Haemophilia, 2020 Haemophilia is a rare X-linked disorder characterized by a deficiency or dysfunction in circulating coagulation factor VIII or IX. Patients with severe haemophilia are at an increased risk of spontaneous bleeding predominantly into the joints ...
Walsh C, Coppens M, Escobar M, Wang M.
europepmc +2 more sources
Eftrenonacog Alfa: A Review in Haemophilia B. [PDF]
Drugs, 2023 Eftrenonacog alfa (Alprolix®) is an extended half-life recombinant factor IX (rFIX)-Fc fusion protein (hereafter referred to as rFIXFc). Administered as an intravenous bolus, it is approved for prophylactic use and the treatment of bleeding in patients with haemophilia B in various countries worldwide, including those of the EU, as well as the USA.
Lamb YN, Hoy SM.
europepmc +3 more sources
Health-related quality of life in paediatric haemophilia B patients treated with rIX-FP. [PDF]
Haemophilia, 2019 Frequent infusions and bleeds can impact on the health‐related quality of life (HRQoL) of paediatric haemophilia B patients. rIX‐FP (IDELVION®) is a fusion protein linking recombinant factor IX with recombinant albumin, and is associated with low ...
von Mackensen S +3 more
europepmc +2 more sources
Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B
Haemophilia, 2010 Summary. Dogs with haemophilia A or haemophilia B exhibit spontaneous bleeding comparable with the spontaneous bleeding phenotype that occurs in humans with severe haemophilia. The phenotypic and genotypic characteristics of haemophilic dogs have been well‐described, and such dogs are suitable for testing prophylactic protein replacement therapy and ...
T C, Nichols +9 more
openaire +4 more sources
Real-World Effectiveness of rFIXFc Prophylaxis in Patients with Haemophilia B Switched from Standard Half-Life Therapy in Three European Countries. [PDF]
Adv Ther, 2023 Funding E +7 more
europepmc +3 more sources
Cost analysis of extended half-life recombinant factor IX products in the treatment of haemophilia B in Italy: an update. [PDF]
Glob Reg Health Technol Assess, 2022 Aiello A +5 more
europepmc +3 more sources