[HTML][HTML] Hemophilia therapy: the future has begun

PM Mannucci - Haematologica, 2020 - ncbi.nlm.nih.gov
The success story of hemophilia care first began in the 1970s, when the availability of
plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided …

Emicizumab: a review in haemophilia A

HA Blair - Drugs, 2019 - Springer
Emicizumab (Hemlibra®), a recombinant, humanized, bispecific monoclonal antibody,
restores the function of missing activated factor VIII (FVIII) by bridging FIXa and FX to …

Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non …

SW Pipe, M Shima, M Lehle, A Shapiro… - The Lancet …, 2019 - thelancet.com
Background Emicizumab, a subcutaneously administered, humanised, bispecific,
monoclonal antibody, is approved to treat people with haemophilia A of all ages with and …

A multicentre, open‐label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors

M Shima, K Nogami, S Nagami, S Yoshida… - …, 2019 - Wiley Online Library
Introduction Emicizumab is a recombinant humanized bispecific monoclonal antibody
mimicking the cofactor function of activated factor VIII. Aim In this multicentre, open‐label …

Pharmacokinetics and pharmacodynamics of emicizumab in persons with hemophilia A with factor VIII inhibitors: HAVEN 1 study

C Schmitt, JI Adamkewicz, J Xu, C Petry… - Thrombosis and …, 2021 - thieme-connect.com
Emicizumab, a bispecific monoclonal antibody, bridges activated factor IX (FIXa) and FX,
replacing the function of missing FVIIIa to restore effective hemostasis in persons with …

Effects and interferences of emicizumab, a humanised bispecific antibody mimicking activated factor VIII cofactor function, on coagulation assays

JI Adamkewicz, DC Chen… - Thrombosis and …, 2019 - thieme-connect.com
Emicizumab bridges activated factor IX (FIX) and FX to restore the tenase function mediated
by activated FVIII (FVIIIa), which is deficient in people with haemophilia A (PwHA). Unlike …

The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab

M Carcao, C Escuriola‐Ettingshausen… - …, 2019 - Wiley Online Library
Introduction As a result of the new treatment paradigm that the haemophilia community will
face with the availability of novel (non‐factor) therapies, an updated consensus on ITI …

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 …

Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors

PT Ebbert, F Xavier, CD Seaman, MV Ragni - Haemophilia, 2020 - Wiley Online Library
Introduction Emicizumab is a bispecific monoclonal antibody that mimics factor VIII (FVIII) by
binding to factors IXa and X to promote hemostasis in haemophilia A (HA) and HA with …

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 …