R. Loeffen +8 more
semanticscholar +1 more source
FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita +2 more
wiley +1 more source
Thrombin generation test as a useful tool for improving disease severity stratification in antiphospholipid syndrome. [PDF]
Yelnik C +9 more
europepmc +1 more source
Gene Editing for Haemophilia—The Next Frontier
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti +3 more
wiley +1 more source
Marstacimab, an antitissue factor pathway inhibitor, combined with bypassing agents: effects on thrombin generation and in hemostatically normal rats. [PDF]
Peraza MA +5 more
europepmc +1 more source
Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community
ABSTRACT Inherited bleeding disorders encompass a diverse group of conditions caused by genetic defects affecting coagulation factors, fibrinogen, von Willebrand factor, or platelet function. Despite major advances in quantitative and functional laboratory assays, a substantial diagnostic gap remains, particularly in patients with mild or atypical ...
Anna R. Blankstein +6 more
wiley +1 more source
ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini +4 more
wiley +1 more source
Measuring FVIII Activity and Thrombin Generation Simultaneously With a Novel Point of Care Platform (EnzySystem HemA): Qualitative Usability Evaluation. [PDF]
Bavinck A +6 more
europepmc +1 more source
ABSTRACT Introduction Monitoring extended half‐life (EHL) factor (F) VIII and FIX products is complex, as discrepant results are obtained in different laboratory assays. Aim To provide an overview on assay accuracy based on a re‐assessment of previously published data as well as laboratory results of EHL spiking experiments for specific factor assays ...
An K. Stroobants +5 more
wiley +1 more source
Altered thrombin generation with prothrombin complex concentrate is not detected by viscoelastic testing: an in vitro study. [PDF]
Hofmann N +5 more
europepmc +1 more source

