Results 41 to 50 of about 29,065 (252)

Preclinical evaluation of a next-generation, subcutaneously administered, coagulation factor IX variant, dalcinonacog alfa.

open access: yesPLoS ONE, 2020
IntroductionThe rapid clearance of factor IX necessitates frequent intravenous administrations to achieve effective prophylaxis for patients with hemophilia B.
Timothy C Nichols   +4 more
doaj   +1 more source

Clinical and Laboratory Characterization of Acquired Von Willebrand Syndrome

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Acquired von Willebrand Syndrome (AVWS) is a rare bleeding disorder characterized by quantitative or qualitative defects of von Willebrand factor (VWF) in patients without a personal or family history of bleeding. It is frequently associated with systemic diseases, particularly lymphoproliferative disorders (LPDs) and myeloproliferative ...
Alessandro Ciavarella   +10 more
wiley   +1 more source

Serum Vitamin D in Children with Hemophilia A and Its Association with Joint Health and Quality of Life

open access: yesHematology Reports
Background/Objectives: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of ...
Aida M. S. Salem   +6 more
doaj   +1 more source

Cardiac surgery in patients with Hemophilia:is it safe?

open access: yesJournal of Cardiothoracic Surgery, 2020
Background The life expectancy of hemophiliacs is similar to that of the general population. As a result, the prevalence of age-related cardiovascular diseases has increased.
Amjad Shalabi   +9 more
doaj   +1 more source

Immune tolerance induction in a severe hemophilia B child with low titer inhibitors

open access: yesJournal of Applied Hematology, 2022
Hemophilia B is an X-linked inherited bleeding disorder caused by either the absence or reduced biosynthesis of clotting factor IX (FIX). This disorder affects approximately 1 in 30,000 male individuals worldwide. Patients with severe form (FIX
Asia Almulla, Najam Awan, Faisl Khanani
doaj   +1 more source

Precision medicine in paediatrics: Progress and priorities

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain   +3 more
wiley   +1 more source

Hemophilia B families with the same mutation are often related a survey of the Swedish population

open access: yes, 2012
Aim: To study if families with hemophilia B in Sweden carrying the same mutation are identical by descent (IBD) or the result of independent mutations (RM).
Kristianstad University.   +3 more
core   +1 more source

Gene Therapy Approaches for the Treatment of Hemophilia B

open access: yes, 2023
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14 days to 2–3 times a week in patients with severe hemophilia B, as a result of a single injection, gene therapy can restore F9 gene expression and maintain
Sofya G. Feoktistova   +3 more
core   +1 more source

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

open access: yesOrphanet Journal of Rare Diseases, 2021
Background Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life.
Tom Burke   +4 more
doaj   +1 more source

The frequency of complications in a cohort of patients diagnosed with hemophilia A and hemophilia B receiving prophylactic treatment in Colombia: A retrospective noninterventional study.

open access: yesPLoS ONE, 2023
IntroductionHemophilia A and B are disorders associated with the deficit of coagulation factors VIII and IX.ObjectiveWas to determine the incidence of complications in a cohort of patients diagnosed with moderate and severe hemophilia A or B under ...
Jorge E Machado Alba   +9 more
doaj   +1 more source

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