Results 51 to 60 of about 82,032 (316)

Report on the diagnosis and treatment of hemophilia in China (Version 2024)

open access: yesLabMed Discovery
Hemophilia is a hemorrhagic disease caused by a lack of clotting factors due to genetic mutations, and is a representative of rare diseases. The prevalence rate of hemophilia is about 2.73/100,000–3.09/100,000 in China, and the absolute number of ...
Feng Xue   +11 more
doaj   +1 more source

Emicizumab Associated Rhabdomyolysis in Hemophilia A

open access: yesClinical Hematology International, 2020
Emicizumab is increasingly the front-line treatment for patients with Hemophilia A with or without inhibitors. Rhabdomyolysis is a syndrome of muscle necrosis and release of intracellular muscle constituents into the circulation.
Joseph A. Wilson   +5 more
doaj   +1 more source

INDUCTION OF TOLERANCE BY ORAL ADMINISTRATION OF FACTOR VIII AND TREATMENT OF HEMOPHILA [PDF]

open access: yes, 2011
Disclosed herein is a simple method for the treatment of antigen-deficiency diseases, by orally administering to a Subject a therapeutically effective amount of the deficient anti gen, wherein the antigen is not present in a liposome.
Alpan, Oral   +3 more
core   +1 more source

Antithrombin: Deficiency, Diversity, and the Future of Diagnostics

open access: yesMass Spectrometry Reviews, EarlyView.
ABSTRACT Our healthcare system provides reactive sick‐care, treating patients after symptoms have appeared by prescription of generic and often suboptimal therapy. This strategy brings along high costs and high pressure which is not sustainable.
Mirjam Kruijt   +2 more
wiley   +1 more source

Porcine model of hemophilia A. [PDF]

open access: yesPLoS ONE, 2012
Hemophilia A is a common X chromosome-linked genetic bleeding disorder caused by abnormalities in the coagulation factor VIII gene (F8). Hemophilia A patients suffer from a bleeding diathesis, such as life-threatening bleeding in the brain and harmful ...
Yuji Kashiwakura   +15 more
doaj   +1 more source

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

open access: yes, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Hemophilia A: An Ideal Disease for Prenatal Therapy

open access: yesPrenatal Diagnosis, EarlyView.
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada   +2 more
wiley   +1 more source

MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA

open access: yesMediterranean Journal of Hematology and Infectious Diseases, 2020
The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino   +2 more
doaj   +1 more source

A Comparison between Recombinant Activated Factor VII (Aryoseven) and Novoseven in Patients with Congenital Factor VII Deficiency [PDF]

open access: yes, 2015
In order to establish the efficacy and biosimilar nature of AryoSeven to NovoSeven in the treatment of congenital factor VII (FVII) deficiency, patients received either agent at 30 1/4g/kg, intravenously per week for 4 weeks, in a randomized fashion. The
Abolghasemi, H.   +16 more
core  

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