Novel therapies for von Willebrand disease

For the past decades, treatment for von Willebrand disease has essentially consisted of classic approaches, and only in the past few years has the need for more innovative strategies been recognized. To address the needs of groups of patients with similar phenotypes and bleeding, personalized therapeutic strategies are being developed, molecules designed for other bleeding disorders are being repositioned, and new hemostatic agents are being tested in patients with von Willebrand disease. New therapeutics include a variety of molecules, including antibodies, engineered factors, synthetic nanoparticles, small interfering RNAs, and genome editing tools. Some promising molecules are still undergoing preclinical testing, whereas others have already entered clinical evaluation and may soon be available for at least some patients with von Willebrand disease. We believe that new approaches will improve the clinical management and the quality of life for patients with von Willebrand Disease.