The era of thromboinflammation: platelets are dynamic sensors and effector cells during infectious diseases

L Guo, MT Rondina - Frontiers in immunology, 2019 - frontiersin.org
Platelets are anucleate cells produced by megakaryocytes. In recent years, a robust body of
literature supports the evolving role of platelets as key sentinel and effector cells in infectious …

Emicizumab, a bispecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII?

PJ Lenting, CV Denis… - Blood, The Journal of the …, 2017 - ashpublications.org
During the last decade, the development of improved and novel approaches for the
treatment of hemophilia A has expanded tremendously. These approaches include factor …

[HTML][HTML] Efanesoctocog alfa prophylaxis for patients with severe hemophilia A

A Von Drygalski, P Chowdary, R Kulkarni… - … England Journal of …, 2023 - Mass Medical Soc
Background Efanesoctocog alfa provides high sustained factor VIII activity by overcoming
the von Willebrand factor–imposed half-life ceiling. The efficacy, safety, and …

Efanesoctocog alfa prophylaxis for children with severe hemophilia A

L Malec, F Peyvandi, AKC Chan… - … England Journal of …, 2024 - Mass Medical Soc
Background Once-weekly efanesoctocog alfa provides high sustained factor VIII activity with
superior bleeding prevention as compared with prestudy factor VIII prophylaxis in previously …

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 …

von Willebrand disease

O Seidizadeh, JCJ Eikenboom, CV Denis… - Nature Reviews …, 2024 - nature.com
Abstract von Willebrand disease (VWD) is the most common inherited bleeding disorder.
The disorder is characterized by excessive mucocutaneous bleeding. The most common …

New therapies for hemophilia

AC Weyand, SW Pipe - Blood, The Journal of the American …, 2019 - ashpublications.org
Hemophilia A (HA) and hemophilia B (HB) are the most common severe bleeding disorders.
Replacement therapy, providing the missing coagulation factor, has been the mainstay of …

[HTML][HTML] BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A

BA Konkle, AD Shapiro, DV Quon… - … England Journal of …, 2020 - Mass Medical Soc
Background Factor VIII replacement products have improved the care of patients with
hemophilia A, but the short half-life of these products affects the patients' quality of life. The …

Advances and innovations in haemophilia treatment

R Peters, T Harris - Nature Reviews Drug Discovery, 2018 - nature.com
Haemophilia is a rare disease for which the approved therapeutic options have remained
virtually unchanged for 50 years. In the past decade, however, there has been an explosion …

BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice

E Seth Chhabra, T Liu, J Kulman… - Blood, The Journal …, 2020 - ashpublications.org
Factor VIII (FVIII) replacement products enable comprehensive care in hemophilia A.
Treatment goals in severe hemophilia A are expanding beyond low annualized bleed rates …