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Hemophilia 

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  1. Open Access 15-03-2024 | Hemophilia | ReviewPaper

    Targeted Gene Insertion: The Cutting Edge of CRISPR Drug Development with Hemophilia as a Highlight

    The remarkable advance in gene editing technology presents unparalleled opportunities for transforming medicine and finding cures for hereditary diseases. Human trials of clustered regularly interspaced short palindromic repeats …

  2. Open Access 15-04-2024 | Hemophilia | Online First

    Comparative Effectiveness of Valoctocogene Roxaparvovec and Prophylactic Factor VIII Replacement in Severe Hemophilia A

    Hemophilia A (HA), a rare X-linked recessive bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII) protein [ 1 ], affects approximately 17.1 per 100,000 male individuals worldwide [ 2 ]. Standard of care (SOC) for individuals …

  3. Open Access 01-12-2024 | Hemophilia | OriginalPaper

    Incidence of deep venous thrombosis in patients with hemophilia undergoing bilateral simultaneous total knee arthroplasty: a retrospective cohort study

    Hemophilia is an inherited bleeding disorder characterized by a deficiency in coagulation factor VIII (hemophilia A) or factor IX (hemophilia B). Recurrent joint bleeding (hemarthroses) can lead to hemophilic arthropathy, which usually affects the …

  4. Open Access 21-03-2024 | Hemophilia | Erratum

    Correction to: Efanesoctocog alfa in hemophilia A: a profile of its use

  5. 13-01-2024 | Hemophilia | Report

    Spontaneous intramural hematoma of the small bowel in a hemophilia  A patient

  6. Open Access 28-03-2024 | Hemophilia | Online First

    In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia

    Hemophilia is a genetic disorder that is caused by mutations in coagulation factor VIII (hemophilia A) or IX (hemophilia B) genes resulting in blood clotting disorders. Despite advances in therapies, such as recombinant proteins and products with …

  7. 13-04-2024 | Hemophilia | Online First

    Cerebral infarction in a patient with inhibitor-positive hemophilia B treated with plasma-derived factor VIIa and factor X mixture

  8. Open Access 24-02-2024 | Hemophilia | ReviewPaper

    Efanesoctocog alfa in hemophilia A: a profile of its use

    Efanesoctocog alfa [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl; ALTUVIIIO®], indicated for routine prophylaxis, on-demand treatment of bleeding episodes, and perioperative management of bleeding, is a useful addition to …

  9. Open Access 07-11-2023 | Hemophilia | OriginalPaper

    Oral health status and oral habits of children and adolescents with hemophilia: a report from the children’s hemophilia comprehensive care center of China

    In recent years, the diagnosis and treatment of hemophilic children in China has significantly improved. However, oral health conditions, which affect quality of life, haven’t received attention in this population. To explore the oral health …

  10. 17-02-2024 | Hemophilia | Letter

    Concomitant large deletion and de novo duplication of factor VIII gene in an Indian patient with severe Hemophilia A

  11. Open Access 01-12-2024 | Hemophilia | OriginalPaper

    Moderate- to vigorous-intensity physical activities for hemophilia A patients during low-dose pharmacokinetic-guided extended half-life factor VIII prophylaxis

    Hemophilia A (HA) is an X-linked recessive congenital bleeding disorder with a deficiency of coagulation factor VIII (FVIII). Patients with severe HA have endogenous FVIII coagulant activity (FVIII:C) levels < 1 IU/dL, making them vulnerable to …

  12. 11-11-2023 | Hemophilia | ReviewPaper

    Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects

    Recent advancements have focused on enhancing factor VIII half-life and refining its delivery methods, despite the well-established knowledge that factor VIII deficiency is the main clotting protein lacking in hemophilia. Consequently, both viral …

  13. 14-02-2024 | IgA Vasculitis | ReviewPaper

    A rare twist: COVID-19 infection masquerading as IgA vasculitis in a hemophilia a patient

    Hemophilia A and B are one of the most common hereditary bleeding disorders. Patients are predisposed to bleeding spontaneously or after minor trauma in different areas such as the skin, gastrointestinal, or joints. COVID-19 infection has been …

  14. Open Access 10-02-2024 | Hemophilia | Letter

    Letter to the Editor Regarding “Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China”

  15. Open Access 10-02-2024 | Hemophilia | Letter

    A Response to: Letter to the Editor Regarding “Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China”

  16. Open Access 17-02-2024 | Hemophilia | OriginalPaper

    Pharmacokinetic–Pharmacodynamic Modelling in Hemophilia A: Relating Thrombin and Plasmin Generation to Factor VIII Activity After Administration of a VWF/FVIII Concentrate

    Hemophilia A is characterized by a deficiency of coagulation factor (F) VIII leading to recurrent spontaneous and trauma-induced bleeding [ 1 ]. Current guidelines recommend treatment with prophylactic FVIII replacement therapy [ 2 ], bypassing …

  17. 03-10-2023 | Hemophilia | Independent Medical Education | Teaser

    Navigating the evolving care of hemophilia patients

    This independent educational program aims to highlight advances in the diagnosis, treatment, and management of patients with hemophilia that have the potential to improve patients’ outcomes and quality of life.

  18. 25-07-2023 | Hemophilia | OriginalPaper

    Successful initiation of hemodialysis for a hemophilia A patient with factor VIII inhibitor: a case report and literature review

    We report the first case of hemophilia A with factor VIII (FVIII) inhibitor who received hemodialysis via an arteriovenous (AV) fistula. Hemophilia A is a congenital deficiency of blood coagulation FVIII that is characterized by prolonged …

  19. Open Access 18-11-2023 | Turner's Syndrome | Case Study

    Hemophilia A and factor V deficiency in a girl with Turner syndrome: a case report

    Hemophilia is rare in females and karyotyping is advised. In this case, a 5-year-old girl who presented with hemorrhage of the gum, epistaxis, and short stature was diagnosed with Turner syndrome, hemophilia A, and factor V deficiency.

  20. 15-01-2024 | Hemophilia | Online First

    Adult People with Hemophilia A Have Low Annualized Bleeding Rate, However the Arthropathy Remains a Burden: A Retrospective Cohort Study

    Congenital Hemophilia A is a complex disease to treat, especially in places without access to hemophilia treatment centers (HTCs). The primary aim of this study was to analyze the outcomes of a cohort of adult people with congenital hemophilia A …

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