Check out the latest articles on von Willebrand disease
Last update : Tuesday March 19, 2024In the last several decades, the bleeding disorders community has seen an unprecedented evolution of novel therapies for treating inherited disorders. These therapies have included recombinant clotting...
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Von Willebrand disease (VWD) is a hemostatic disorder characterized by a quantitative or qualitative deficiency of the Von Willebrand factor (VWF). It affects males and females equally. This pathology...
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In order for patients to gain access to new pharmaceuticals, manufacturers must successfully navigate two critical steps. First, they need to obtain marketing approval (i.e., approval to market) from a...
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Patients with an unexplained mild to moderate bleeding tendency are diagnosed with bleeding disorder of unknown cause (BDUC), a classification reached after ruling out other mild to moderate bleeding disorders (MBD) including von Willebrand disease (VWD), platelet function defects (PFDs), coagulation factor deficiencies (CFDs), and non-hemostatic causes for bleeding. This review outlines our diagnostic approach to BDUC, a diagnosis of exclusion, drawing...
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1 Real-world evidence for coverage determination of treatments for rare diseases
Orphanet Journal of Rare Diseases 2024; 19(1): 47