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Unbound Medicine.
(Hemophilia B)
5,349 results
  • Gene Therapy for Inherited Bleeding Disorders. [Journal Article]
    Semin Thromb Hemost. 2021 Mar; 47(2):161-173.Arruda VR, Weber J, Samelson-Jones BJ
  • Decades of preclinical and clinical studies developing gene therapy for hemophilia are poised to bear fruit with current promising pivotal studies likely to lead to regulatory approval. However, this recent success should not obscure the multiple challenges that were overcome to reach this destination. Gene therapy for hemophilia A and B benefited from advancements in the general gene therapy fie…
  • Novel treatments for hemophilia through rebalancing of the coagulation cascade. [Review]
    Pediatr Blood Cancer. 2021 Feb 12 [Online ahead of print]Zhao Y, Weyand AC, Shavit JA
  • Hemophilia A and B are inherited hemorrhagic disorders that result from alterations in the coagulation cascade. Aside from spontaneous bleeding, the main complication of hemophilia is hemarthrosis. Progress over the last three decades, specifically prophylaxis using recombinant factor, has prevented hemarthrosis and lengthened patient life expectancies. However, many treatments require frequent d…
  • Concizumab: a novel anti-TFPI therapeutic for hemophilia. [Journal Article]
    Blood Adv. 2021 Jan 12; 5(1):279.Shapiro AD
  • Concizumab is a novel subcutaneous prophylactic therapy for hemophilia. It is a hemostatic rebalancing agent that binds to the Kunitz-2 domain of tissue factor pathway inhibitor (TFPI), one of the molecules that contributes to downregulation of coagulation thereby preventing TFPI from binding to and blocking the factor Xa (FXa) active site. When the TFPI inhibitory activity is decreased, sufficie…
  • Functional and joint evaluation in a prospective cohort of patients with severe haemophilia. [Journal Article]
    Haemophilia. 2021 Feb 06 [Online ahead of print]Ribeiro AJT, Amorim FF, … Imoto AM
  • CONCLUSIONS: The older the patient with haemophilia, the higher the probability of a worsening of the HJHS. In the presence of more arthropathies, the older the patient, the worse the FISH. Patients receiving primary prophylaxis show better results in the HJHS and FISH when compared to patients receiving secondary prophylaxis and/or on-demand treatment.
  • Current challenges for men and women with mild-to-moderate haemophilia. [Journal Article]
    Haemophilia. 2021 Jan; 27 Suppl 1:5-7.Rajpurkar M, Forsyth A, Manco-Johnson M
  • Current treatments in the field of haemophilia are changing the phenotype of many patients with severe haemophilia to that of mild haemophilia. Despite this improvement, those with mild-to-moderate haemophilia A and haemophilia B continue to experience unmet needs. Whereas some patients with mild-to-moderate haemophilia experience similar complications to those of patients with severe haemophilia…
  • Mechanisms of Bone Remodeling Disorder in Hemophilia. [Journal Article]
    Semin Thromb Hemost. 2021 Feb; 47(1):43-52.Wang H, Bai X
  • Hemophilia is caused by a lack of antihemophilic factor(s), for example, factor VIII (FVIII; hemophilia A) and factor IX (FIX; hemophilia B). Low bone mass is widely reported in epidemiological studies of hemophilia, and patients with hemophilia are at an increased risk of fracture. The detailed etiology of bone homeostasis imbalance in hemophilia is unclear. Clinical and experimental studies sho…
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