Search Results - "Quon, Doris V"
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BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A
Published in The New England journal of medicine (10-09-2020)“…In a study involving patients with severe hemophilia A, an injection of the novel fusion protein BIVV001 resulted in a duration of factor VIII activity that…”
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Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A
Published in Journal of thrombosis and haemostasis (01-07-2024)“…Valoctocogene roxaparvovec transfers a human factor (F)VIII coding sequence into hepatocytes of people with severe hemophilia A to provide bleeding protection…”
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Prevalence and Risk Factors for Hypertension in Hemophilia
Published in Hypertension (Dallas, Tex. 1979) (01-07-2013)“…Hypertension (HTN) is a major risk factor for intracranial hemorrhage. We, therefore, investigated the prevalence, treatment, and control of HTN in adult…”
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Consistency of serial ultrasonographic joint tissue measurements by the Joint tissueActivity and Damage Exam (JADE) protocol in relation to hemophilic joint health parameters
Published in BMC musculoskeletal disorders (15-04-2023)“…Abstract Objectives The Joint tissue Activity and Damage Exam (JADE) is a point-of-care (POC) musculoskeletal ultrasound (MSKUS) protocol for non-radiologists…”
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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
Published in The New England journal of medicine (17-03-2022)“…Patients with severe hemophilia A were treated with an adenoviral construct containing coagulation factor VIII cDNA and followed for 1 to 3 years. Median…”
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Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
Published in The New England journal of medicine (23-02-2023)“…Participants with hemophilia A received B-domain–deleted factor VIII gene therapy delivered in an AAV5 vector. A decrease in annualized bleeding rates was…”
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The Hypertension of Hemophilia Is Not Explained by the Usual Cardiovascular Risk Factors: Results of a Cohort Study
Published in International journal of hypertension (01-01-2016)“…Background. The etiology of the high prevalence of hypertension among patients with hemophilia (PWH) remains unknown. Methods. We compared 469 PWH in the…”
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Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A
Published in Human gene therapy (01-04-2022)“…Adeno-associated virus (AAV)-mediated gene therapy may provide durable protection from bleeding events and reduce treatment burden for people with hemophilia A…”
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Invasive procedures and surgery following etranacogene dezaparvovec gene therapy in people with hemophilia B
Published in Journal of thrombosis and haemostasis (26-09-2024)“…Little information regarding the management of invasive procedures in people with hemophilia B (HB) after undergoing gene therapy is available. Here, we report…”
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Real‐world data demonstrate improved bleed control and extended dosing intervals for patients with haemophilia B after switching to recombinant factor IX Fc fusion protein (rFIXFc) for up to 5 years
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01-11-2020)“…Introduction In clinical trials, recombinant factor IX fusion protein (rFIXFc) has demonstrated safety, efficacy and prolonged activity with extended dosing…”
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Risk of diabetes in haemophilia patients compared to clinic and non‐clinic control cohorts
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01-05-2022)“…Introduction Ageing patients with haemophilia (PWH) develop cardiovascular risk factors impacting care. Little is known about the prevalence of diabetes in PWH…”
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Development and Reliability of the Joint Tissue Activity and Damage Examination for Quantitation of Structural Abnormalities by Musculoskeletal Ultrasound in Hemophilic Joints
Published in Journal of ultrasound in medicine (01-06-2019)“…Objectives Musculoskeletal ultrasound (US) is used increasingly to examine hemophilic arthropathy. However, quantitative algorithms to document findings are…”
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Blood pressure in persons with haemophilia with a focus on haemophilia‐specific risk factors
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01-11-2022)“…Introduction Persons with haemophilia (PWH) have a higher prevalence of hypertension compared to the general population, which cannot be explained entirely by…”
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Diabetes, hepatitis C and human immunodeficiency virus influence hypertension risk differently in cohorts of haemophilia patients, veterans and the general population
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01-11-2022)“…Introduction The reasons for the high prevalence of hypertension in persons with haemophilia (PWH) are poorly understood. Aim To examine the roles of diabetes,…”
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Physical activity in individuals with haemophilia and experience with recombinant factor VIII Fc fusion protein and recombinant factor IX Fc fusion protein for the treatment of active patients: a literature review and case reports
Published in Blood coagulation & fibrinolysis (01-10-2016)“…The World Federation of Hemophilia and the National Hemophilia Foundation encourage people with haemophilia (PWH) to participate in routine physical activity…”
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A Dynamin Is Required for the Biogenesis of Secretory Organelles in Toxoplasma gondii
Published in Current biology (24-02-2009)“…Apicomplexans contain only a core set of factors involved in vesicular traffic. Yet these obligate intracellular parasites evolved a set of unique secretory…”
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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia
Published in Expert review of hematology (15-03-2023)“…Decades of research have transformed hemophilia from severely limiting children's lives to a manageable disorder compatible with a full, active life, for many…”
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A phase 1B/2 study of aldoxorubicin in patients with soft tissue sarcoma
Published in Cancer (15-02-2015)“…BACKGROUND Aldoxorubicin, a prodrug of doxorubicin, covalently binds to serum albumin, allowing for the administration of much higher doses of doxorubicin in a…”
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Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
Published in The Lancet. Haematology (01-04-2024)“…Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophylactic…”
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A Prospective Observational Study of Antihemophilic Factor (Recombinant) Prophylaxis Related to Physical Activity Levels in Patients with Hemophilia A in the United States (SPACE)
Published in Journal of blood medicine (01-01-2021)“…High collision-risk physical activity can increase bleeding risk in people with hemophilia A, as can increasing the time between factor VIII (FVIII)…”
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