Search Results - "Doshi, Bhavya S"
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Novel approaches to hemophilia therapy: successes and challenges
Published in Blood (23-11-2017)“…New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to nonfactor products and…”
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AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life
Published in Molecular therapy. Methods & clinical development (14-03-2024)“…The hemophilias are the most common severe inherited bleeding disorders and are caused by deficiency of clotting factor (F) VIII (hemophilia A) or FIX…”
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Emerging therapies for hemophilia: controversies and unanswered questions
Published in F1000 research (2018)“…Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor…”
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Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog
Published in Molecular therapy. Methods & clinical development (14-12-2023)“…Adeno-associated viral (AAV) vectors have traditionally been viewed as predominantly nonintegrating, with limited concerns for oncogenesis. However,…”
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Potentiation of thrombin generation in hemophilia A plasma by coagulation factor VIII and characterization of antibody-specific inhibition
Published in PloS one (29-10-2012)“…Development of inhibitory antibodies to coagulation factor VIII (fVIII) is the primary obstacle to the treatment of hemophilia A in the developed world. This…”
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RNAing toward a new therapy for hemophilia
Published in Blood (30-05-2024)Get full text
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Coagulation factor VIII: biological basis of emerging hemophilia A therapies
Published in Blood (01-08-2024)“…[Display omitted] Coagulation factor VIII (FVIII) is essential for hemostasis. After activation, it combines with activated FIX (FIXa) on anionic membranes to…”
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Gene therapy for hemophilia: what does the future hold?
Published in Therapeutic Advances in Hematology (01-09-2018)“…Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have reported remarkable success in ameliorating disease phenotype in…”
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Combined anti‐CD20 and mTOR inhibition with factor VIII for immune tolerance induction in hemophilia A patients with refractory inhibitors
Published in Journal of thrombosis and haemostasis (01-04-2020)“…Background Hemophilia A (HA) inhibitor patients that fail traditional immune tolerance induction (ITI) have increased morbidity and mortality. Preclinical…”
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Gene therapy for hemophilia: Progress to date and challenges moving forward
Published in Transfusion and apheresis science (01-10-2019)“…Over the past decades hemophilia has been transformed from a debilitating disease to a manageable condition. However, the current treatment options are…”
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Hemolysis After Medication Exposure in Pediatric Patients With G6PD Deficiency
Published in Journal of pediatric hematology/oncology (01-07-2022)“…Hemolysis in glucose-6-phosphate dehydrogenase (G6PD) deficiency varies by mutation status and the oxidative stressor. Although classified by percent of…”
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Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab
Published in Blood (12-09-2024)“…Emicizumab improves the procoagulant activity of select loss-of-function factor IX (FIX) variants with likely dysfunctional assembly of the intrinsic Xase…”
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Gene Therapy for Hemophilia: Facts and Quandaries in the 21st Century
Published in Mediterranean journal of hematology and infectious diseases (01-09-2020)“…Therapy for hemophilia has evolved in the last 40 years from plasma-based concentrates to recombinant proteins and, more recently, to non-factor therapeutics…”
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Diagnosis and Management of Pediatric Venous Thromboembolism: New Therapies on the Horizon
Published in Pediatric emergency care (01-05-2021)“…The incidence of venous thromboembolism (VTE) is increasing in pediatric patients. Prompt recognition and evaluation of VTE in young patients could prevent…”
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B cell-activating factor modulates the factor VIII immune response in hemophilia A
Published in The Journal of clinical investigation (15-04-2021)“…Inhibitors of factor VIII (FVIII) remain the most challenging complication of FVIII protein replacement therapy in hemophilia A (HA). Understanding the…”
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Coagulation factor VIII regulates von Willebrand factor homeostasis invivo
Published in Journal of thrombosis and haemostasis (01-12-2023)“…Coagulation factor VIII (FVIII) and von Willebrand factor (VWF) circulate as a noncovalent complex, but each has its distinct functions. Binding of FVIII to…”
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Utility of repeat testing in the evaluation for von Willebrand disease in pediatric patients
Published in Journal of thrombosis and haemostasis (01-11-2019)“…Background Von Willebrand disease (VWD) is the most common inherited bleeding disorder and is caused by quantitative and qualitative defects in von Willebrand…”
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A major determinant of the immunogenicity of factor VIII in a murine model is independent of its procoagulant function
Published in Blood (20-09-2012)“…A main complication of treatment of patients with hemophilia A is the development of anti–factor VIII (fVIII) antibodies. The immunogenicity of fVIII…”
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Iliopsoas hematoma in a patient with sickle cell disease
Published in Pediatric blood & cancer (01-07-2018)“…Although musculoskeletal pain in patients with sickle cell disease (SCD) is most frequently the result of vaso‐occlusive episodes, clinicians often consider…”
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