The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing
ME Mancuso, JN Mahlangu, SW Pipe - The Lancet, 2021 - thelancet.com
Congenital haemophilia A (factor VIII deficiency) and B (factor IX deficiency) are X-linked
bleeding disorders. Replacement therapy has been the cornerstone of the management of …
bleeding disorders. Replacement therapy has been the cornerstone of the management of …
Current and future therapies for haemophilia—Beyond factor replacement therapies
K Nogami, M Shima - British journal of haematology, 2023 - Wiley Online Library
Some non‐factor products that work by facilitating the coagulation pathway (emicizumab)
and blocking the anticoagulant pathway (fitusiran, concizumab and marstacimab) for …
and blocking the anticoagulant pathway (fitusiran, concizumab and marstacimab) for …
[HTML][HTML] Phase 3 trial of concizumab in hemophilia with inhibitors
Background Concizumab is an anti–tissue factor pathway inhibitor monoclonal antibody
designed to achieve hemostasis in all hemophilia types, with subcutaneous administration …
designed to achieve hemostasis in all hemophilia types, with subcutaneous administration …
In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy
JP Han, MJ Kim, BS Choi, JH Lee, GS Lee, M Jeong… - Science …, 2022 - science.org
Hemophilia is a hereditary disease that remains incurable. Although innovative treatments
such as gene therapy or bispecific antibody therapy have been introduced, substantial …
such as gene therapy or bispecific antibody therapy have been introduced, substantial …
Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors
AD Shapiro, P Angchaisuksiri, J Astermark… - Blood …, 2022 - ashpublications.org
Despite current therapies, there remains an unmet need for treatment for patients with
hemophilia. The main parts of two phase 2 trials established clinical proof-of-concept for …
hemophilia. The main parts of two phase 2 trials established clinical proof-of-concept for …
A molecular revolution in the treatment of hemophilia
JSS Butterfield, KM Hege, RW Herzog, R Kaczmarek - Molecular Therapy, 2020 - cell.com
For decades, the monogenetic bleeding disorders hemophilia A and B (coagulation factor
VIII and IX deficiency) have been treated with systemic protein replacement therapy. Now …
VIII and IX deficiency) have been treated with systemic protein replacement therapy. Now …
Advances in the management of haemophilia: emerging treatments and their mechanisms
D Okaygoun, DD Oliveira, S Soman… - Journal of biomedical …, 2021 - Springer
Mainstay haemophilia treatment, namely intravenous factor replacement, poses several
clinical challenges including frequent injections due to the short half-life of recombinant …
clinical challenges including frequent injections due to the short half-life of recombinant …
Recent advances in the treatment of hemophilia: a review
E Marchesini, M Morfini, L Valentino - Biologics: Targets and …, 2021 - Taylor & Francis
Progress in hemophilia therapy has been remarkable in the first 20 years of the third
millennium, but the innovation began with the description the fractionation of plasma in …
millennium, but the innovation began with the description the fractionation of plasma in …
Long‐term safety and efficacy of the anti‐tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: phase II study results
J Mahlangu, J Luis Lamas… - British Journal of …, 2023 - Wiley Online Library
Marstacimab, an investigational human monoclonal antibody targeting tissue factor pathway
inhibitor, demonstrated safety and efficacy in preventing bleeding episodes in patients with …
inhibitor, demonstrated safety and efficacy in preventing bleeding episodes in patients with …
[HTML][HTML] Regulation of coagulation by tissue factor pathway inhibitor: Implications for hemophilia therapy
AE Mast, W Ruf - Journal of Thrombosis and Haemostasis, 2022 - Elsevier
Tissue factor pathway inhibitor (TFPI) is an alternatively spliced anticoagulant protein that
primarily dampens the initiation phase of coagulation before thrombin is generated. As such …
primarily dampens the initiation phase of coagulation before thrombin is generated. As such …