Results 111 to 120 of about 22,643,381 (355)
Diagnosis of von Willebrand disease in Argentina: a single institution experience [PDF]
von Willebrand disease (VWD) is the most common autosomal bleeding disorder, mostlyinherited as dominant trait. VWD is due to deficiency/abnormality of von Willebrand factor (VWF).
Blanco, Alicia Noemi+5 more
core +1 more source
Catheter Ablation for Atrial Fibrillation in Patients with Hemophilia or von Willebrand Disease
Background Management of atrial fibrillation (AF) is complex in patients with bleeding disorders. Catheter ablation such as pulmonary vein isolation (PVI) has been suggested in cases with bleeding disorders. However, data on safety are missing.
Paul R. van der Valk+3 more
doaj +1 more source
An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B
Fitusiran is an RNA interference therapeutic that targets antithrombin (AT) in the liver and interferes with AT translation by binding and degrading messenger RNA-AT, thereby silencing AT gene expression and preventing AT synthesis.
Nicoletta Machin, M. Ragni
semanticscholar +1 more source
Antithrombin: Deficiency, Diversity, and the Future of Diagnostics
ABSTRACT Our healthcare system provides reactive sick‐care, treating patients after symptoms have appeared by prescription of generic and often suboptimal therapy. This strategy brings along high costs and high pressure which is not sustainable.
Mirjam Kruijt+2 more
wiley +1 more source
Timing of inhibitor development in >1000 previously untreated patients with severe hemophilia A.
TO THE EDITOR: Inhibitory antibodies (inhibitors) against factor VIII (FVIII) develop in 25% to 35% of previously untreated patients (PUPs) with severe hemophilia A (SHA). It is the most serious complication of classic hemophilia treatment.[1][1][⇓][2]-[
H. M. van den Berg+9 more
semanticscholar +1 more source
Abstract Objective Evaluate the use of tranexamic acid (TXA) and observation as a management option for pediatric patients presenting with posttonsillectomy hemorrhage (PTH). Study Design Retrospective analysis of a prospectively implemented quality improvement initiative with a historical control comparison group. Setting Tertiary children's hospital.
Laura A. Petrauskas+12 more
wiley +1 more source
Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII
Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as FVIII protein injection and gene therapies via AAV-delivered F8 transgene in
Hainan Chen+10 more
semanticscholar +1 more source
ABSTRACT Background Direct oral anticoagulants (DOACs) have quickly gained favor due to oral availability, favorable risk profile, and reduced lab monitoring. This study aims to evaluate trends in DOAC use among pediatric patients with new venous thrombotic events (VTE) and factors associated with their selection.
Molly Mack+2 more
wiley +1 more source
The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B.
Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
M. Soucie+4 more
semanticscholar +1 more source
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa+4 more
wiley +1 more source