Results 1 to 10 of about 113,823 (359)

A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH

Journal of Thrombosis and Haemostasis, 2021
Hemophilia A and B predominantly attracts clinical attention in males due to X‐linked inheritance, introducing a bias toward female carriers to be asymptomatic.
Karin P M Van Galen, Roseline d'Oiron, Peter A Kouides   +2 more
exaly   +2 more sources

Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A [PDF]

Journal of Thrombosis and Haemostasis, 2015
This work was supported by funding from Biogen, including funding for the editorial and writing support in the the development of this ...
Allen, G, Barnes, C, Cristiano, LM, Gambino, G, Kulkarni, R, Liesner, R, Mahlangu, J, Neelakantan, S, Nolan, B, Pasi, J, Pierce, GF, Young, G   +11 more
exaly   +3 more sources

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

Blood, 2021
In the past decade enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered AAV-based liver-directed gene therapy in patients with severe hemophilia B ...
Frank Leebeek
exaly   +2 more sources

Update on clinical gene therapy for hemophilia

Blood, 2019
In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are currently in clinical development, gene therapy holds the promise of a lasting cure with a single drug administration. Near-to-complete correction of hemophilia
Roland W Herzog, David M Markusic
exaly   +2 more sources

Guidelines for the management of hemophilia

Haemophilia, 2013
A. Srivastava, A. K. Brewer, E. Mauser-Bunschoten, N. Key, S. Kitchen, A. Llinás, C. A. Ludlam, Johnny Mahlangu, K. Mulder, M. Poon, A. Street   +10 more
exaly   +2 more sources

Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia

New England Journal of Medicine, 2007
Sharon Funk Dpt, Michael Recht, J Michael Soucie   +2 more
exaly   +2 more sources

Thrombotic diseases and conditions – diagnosis and monitoring of anticoagulant therapy

Медицинский совет, 2021
Laboratory methods are a very important part of the examination of patients with thrombotic diseases, often putting the final touches on the diagnosis, and in some cases even defining this diagnosis.
A. L. Melkumyan, A. L. Berkovskiy, S. A. Vasiliev, E. V. Sergeeva   +3 more
doaj   +1 more source

Women with Hemophilia: Case Series of Reproductive Choices and Review of Literature

TH Open, 2021
Aim Very little is known regarding reproductive choices, pregnancy, and delivery of women with moderate to severe hemophilia. Our aim was to describe our experience with three hemophiliac women and their journey to achieve motherhood.
Shadan Lalezari, Assaf A. Barg, Rima Dardik, Jacob Luboshitz, Dalia Bashari, Einat Avishai, Gili Kenet   +6 more
doaj   +1 more source

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.

New England Journal of Medicine, 2023
BACKGROUND Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against bleeding ...
S. Pipe, F. Leebeek, M. Recht, N. Key, G. Castaman, Wolfgang A. Miesbach, S. Lattimore, K. Peerlinck, P. R. van der Valk, M. Coppens, P. Kampmann, K. Meijer, Niamh O'Connell, K. Pasi, D. Hart, R. Kazmi, J. Astermark, C. Hermans, R. Klamroth, Richard W. Lemons, N. Visweshwar, Annette von Drygalski, G. Young, S. Crary, M. Escobar, E. Gomez, R. Kruse-Jarres, D. Quon, E. Symington, Michael Wang, Allison P. Wheeler, R. Gut, Yinyin Liu, R. Dolmetsch, D. Cooper, Yanyan Li, B. Goldstein, P. Monahan   +37 more
semanticscholar   +1 more source

Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.

New England Journal of Medicine, 2023
BACKGROUND Efanesoctocog alfa provides high sustained factor VIII activity by overcoming the von Willebrand factor-imposed half-life ceiling. The efficacy, safety, and pharmacokinetics of efanesoctocog alfa for prophylaxis and treatment of bleeding ...
Annette von Drygalski, P. Chowdary, R. Kulkarni, S. Susen, B. Konkle, J. Oldenburg, Davide Matino, R. Klamroth, A. Weyand, V. Jiménez‐Yuste, K. Nogami, S. Poloskey, B. Winding, A. Willemze, K. Knobe   +14 more
semanticscholar   +1 more source