RareWays is a research directory. It helps you understand the science. It is not medical advice. Always discuss your care options with your healthcare team.
Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Von Willebrand Disease: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Small interfering RNA targeting protein Z-dependent protease inhibitor improves hemostasis in Von Willebrand factor- and Factor X-deficient mice.
Hamze Rim et al., Thrombosis and haemostasis (14 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42874635/
- 2.
Cost comparison of pdVWF/FVIII prophylaxis and on-demand therapy in type 3 von Willebrand disease in the United States.
Sidonio Robert F et al., Blood advances (13 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42431629/
- 3.
100 Years of von Willebrand Disease (VWD): Comparison of Historical and Contemporary von Willebrand Factor (VWF) Activity Assays in the Laboratory Identification of VWD.
Favaloro Emmanuel J et al., Haemophilia : the official journal of the World Federation of Hemophilia (12 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42864839/
- 4.
Von Willebrand Disease (VWD) 100 years after its first description: the research of Italian investigators for a better management of VWD patients
Augusto Bramante Federici et al., Bleeding Thrombosis and Vascular Biology (9 October 2026)
https://doi.org/10.4081/btvb.2026.580
- 5.
The Contribution of Bleeding Assessment Score and Pictorial Blood Loss Assessment Chart to the Diagnosis of Von Willebrand Disease in Adolescent Girls With Iron Deficiency Anemia.
Turgutoglu Yilmaz Asli et al., Journal of pediatric hematology/oncology (8 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42848004/
Clinical Trials : Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)
NCT07115004 · Recruiting · Phase 3 · Vega Therapeutics, Inc
Ages 12 to 75
https://clinicaltrials.gov/study/NCT07115004
- 2.
A Study Assessing HMB-002 in Participants With Von Willebrand Disease
NCT06754852 · Recruiting · Phase 1 / Phase 2 · Hemab ApS
Ages 16 to 69
https://clinicaltrials.gov/study/NCT06754852
- 3.
Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)
NCT05776069 · Recruiting · Phase 1 / Phase 2 · Vega Therapeutics, Inc
Ages 12 to 60
https://clinicaltrials.gov/study/NCT05776069
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Von Willebrand Disease
Von Willebrand Disease is the most common inherited bleeding disorder, caused by a deficiency or dysfunction of von Willebrand factor, a protein essential for blood clotting. It affects approximately 1% of the global population, though most cases are mild. Symptoms include easy bruising, prolonged bleeding from cuts, and heavy menstrual periods.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
Non-factor therapeutics have transformed the treatment paradigm in hemophilia A. Owing to their mode of action, these agents may also provide benefit for other rare bleeding disorders (RBDs) such as von Willebrand disease (VWD)-type 3 and Factor X (FX) deficiency. Targeting protein Z-dependent protease inhibitor (ZPI), a natural anticoagulant, is a novel strategy to enhance hemostasis. We evaluated the effects of ZPI post-transcriptional silencing using a GalNAc-conjugated siRNA (mTGM-148) in von Willebrand factor-deficient (VwfKO) mice and in mice with strongly reduced factor X levels (< 1%; f10low-mice). mTGM-148 reduced plasma ZPI antigen below 15% in both models. In VwfKO-mice, mTGM-148 significantly enhanced contact activation-initiated thrombin generation and normalized INTEM clotting parameters. In f10low-mice, thrombin generation was minimally affected, but EXTEM clotting parameters were significantly ameliorated. Importantly, mTGM-148 treatment significantly reduced blood loss in the tail clip model for both VwfKO-mice and f10low-mice. These data suggest that ZPI post-transcriptional silencing is a viable strategy to improve the hemostatic potential in both VWD-type 3 and FX deficiency. As such, ZPI inhibition represents a promising non-factor therapeutic approach for selected RBDs.
Common Questions
What is Von Willebrand Disease?
Von Willebrand Disease is the most common inherited bleeding disorder, caused by a deficiency or dysfunction of von Willebrand factor, a protein essential for blood clotting. It affects approximately 1% of the global population, though most cases are mild. Symptoms include easy bruising, prolonged bleeding from cuts, and heavy menstrual periods.
How many clinical trials are available for Von Willebrand Disease?
RareWays currently indexes 49 clinical trials for Von Willebrand Disease, of which 13 are actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Von Willebrand Disease come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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