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The future of siRNA-mediated approaches to treat von Willebrand disease.

Expert Rev Hematol

January 2025

Department of Internal Medicine, Division of Thrombosis and Hemostasis, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands.

Introduction: The clinical management of the inherited bleeding disorder von Willebrand disease (VWD) focuses on normalizing circulating levels of von Willebrand factor (VWF) and factor VIII (FVIII) to prevent or control bleeding events. The heterogeneous nature of VWD, however, complicates effective disease management and development of universal treatment guidelines.

Areas Covered: The current treatment modalities of VWD and their limitations are described and why this prompts the development of new treatment approaches.

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Diabetes insipidus (DI) is a rare endocrine disease involving antidiuretic hormone (ADH), encompassing both central and nephrogenic causes. Inability to respond to or produce ADH leads to inability of the kidneys to reabsorb water, resulting in hypotonic polyuria and, if lack of hydration, hypernatremia. DI cannot be cured and is an unfamiliar disease process to many clinicians.

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[Lower urinary tract dysfunction in children].

Urologie

January 2025

Klinik für Urologie und Kinderurologie, Uniklinik RWTH Aachen, Pauwelsstr. 30, 52074, Aachen, Deutschland.

Lower urinary tract dysfunction (LUTD) is common in children and can significantly impact the quality of life in affected children and their families. This article provides a comprehensive overview of the causes, diagnostics, and treatment, with a particular focus on nonorganic urinary incontinence and nocturnal enuresis. Accurate diagnostics are essential to differentiate between organic and functional causes, as well as to distinguish primary from secondary forms of urinary incontinence.

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: Arginine vasopressin deficiency (AVP-D) is a common condition in the setting of brain death. The aim of this study was to analyze the frequency of AVP-D in organ donors, its treatment, as well as the impact of AVP-D on hemodynamics and renal function. : This single-center, retrospective study included 63 organ donors treated between 2017 and 2022.

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Background: Hemorrhagic stroke (HS) is associated with high risk of mortality or disability. To date, up to 25% of HSs are associated with anticoagulants and antiplatelet agents. Early hemostatic therapy and correction of effect of antithrombotic drugs in patients with HS significantly reduce the risk of adverse outcomes.

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