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Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich+7 more
doaj
Hemophilia A gene therapy: current and next-generation approaches
Introduction Hemophilia comprises a group of X-linked hemorrhagic disorders that result from a deficiency of coagulation factors. The disorder affects mainly males and leads to chronic pain, joint deformity, reduced mobility, and increased mortality ...
S. Pipe, Gil Gonen-Yaacovi, O. Segurado
semanticscholar +1 more source
Hemophilia: a biography on therapeutical approaches
The history of hemophilia is ancient, with descriptions dated to the 2nd century AD. The first modern narratives appeared in 1800s, when total blood transfusion was the only available treatment and life expectancy was remarkably low.
Dayenne Catelli+7 more
doaj
Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors
BACKGROUND Emicizumab is a bispecific monoclonal antibody that bridges activated factor IX and factor X to replace the function of missing activated factor VIII, thereby restoring hemostasis.
J. Mahlangu+16 more
semanticscholar +1 more source
BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.
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 half-life of recombinant factor VIII ranges from 15 to 19 hours because
B. Konkle+11 more
semanticscholar +1 more source
Randall Curtis,1 Marilyn Manco-Johnson,2 Barbara A Konkle,3 Roshni Kulkarni,4 Joanne Wu,5 Judith R Baker,6 Megan Ullman,7 Duc Quang Tran Jr,8 Michael B Nichol5 1Factor VIII Computing, Berkeley, CA, USA; 2Hemophilia and Thrombosis Center, University of ...
Curtis R+8 more
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Factor VIII (FVIII) replacement products enable comprehensive care in hemophilia A. Treatment goals in severe hemophilia A are expanding beyond low annualized bleed rates to include long-term outcomes associated with high sustained FVIII levels.
Ekta Seth Chhabra+29 more
semanticscholar +1 more source
Hemophilia management: Huge impact of a tiny difference
Hemophilia A and B are inherited X‐linked disorders of hemostasis, associated with an increased bleeding tendency. Patients with severe hemophilia have undetectable clotting factor levels and experience spontaneous bleeds.
Fabienne Kloosterman+5 more
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A ten-week-old male Golden retriever puppy was presented with severe hyperesthesia, mild neurological deficits and episcleral bleeding. Clotting times showed a normal prothrombin time (PT) and prolonged activated partial thromboplastin time (aPPT ...
Charlotte Lubbers+3 more
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"Prevalence of inhibitors in a population of 1280 Hemophilia A patients in Iran " [PDF]
Development of inhibitor to factor VIII is the most serious complication of hemophilia therapy. To determine the prevalence of inhibitors in Iran hemophilia A patients exposed to blood products, 1280 hemophilia A patients (age range 9 months-84 years ...
Sharifian R+6 more
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