Materiały źródłowe

• #1 Von Willebrand Disease Epidemiology, Burden of Illness and Management: A Systematic Review

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9987238/

  Although hereditary von Willebrand disease (VWD) is the most common bleeding disorder, its epidemiology is not well understood. […] This systematic review was, therefore, conducted to better understand the disease and related unmet needs for the patients. Data on the epidemiology of VWD worldwide were reviewed, including incidence, prevalence, mortality, patient characteristics, natural history, burden of illness, and therapeutic management/treatments received in the real-world clinical practice setting. […] Available data suggest that patients with VWD experience high disease burden in terms of bleeding, poor quality of life, and health care resource utilization. […] Estimates of the prevalence of VWD (overall or by specific type) were available from 22 of the 168 sources and varied according to the method of estimation.

• #2 Von Willebrand Disease Epidemiology, Illness and Management | JBM

  https://www.dovepress.com/von-willebrand-disease-epidemiology-burden-of-illness-and-management-a-peer-reviewed-fulltext-article-JBM

  Although hereditary von Willebrand disease (VWD) is the most common bleeding disorder, its epidemiology is not well understood. […] A systematic review on the epidemiology/burden of illness of VWD was conducted to better understand patients unmet needs. […] Reported VWD prevalence (22 sources) ranged from 108.9 to 2200 per 100,000 in population-based studies and from 0.3 to 16.5 per 100,000 in referral-based studies. […] Available data suggest that patients with VWD experience high disease burden in terms of bleeding, poor quality of life, and health care resource utilization. […] This systematic review shows that there are limited data available on the epidemiology of VWD, indicating the need for more research to be conducted. […] VWD prevalence estimates varied highly according to the study populations and method of estimation.

• #3 Von Willebrand Disease (VWD) | Symptoms & Treatments | NBDF

  https://www.bleeding.org/bleeding-disorders-a-z/types/von-willebrand-disease

  3200000 or 3.2 million people in the US […] VWD is the most common bleeding disorder, affecting up to 1% of the US population or approximately 1 in every 100 people. […] VWD occurs equally across all races and ethnicities. […] The guidelines for VWD were updated in 2021. […] New VWD Guidelines for the diagnosis and management of von Willebrand disease were released in January 2021. These updated guidelines are the result of a collaborative effort between the American Society of Hematology (ASH), the International Society on Thrombosis and Hemostatsis (ISTH), the National Bleeding Disorders Foundation (NBDF), and the World Federation for Hemophilia (WFH).

• #4 Von Willebrand disease – Knowledge @ AMBOSS

  https://www.amboss.com/us/knowledge/von-willebrand-disease/

  Most common congenital bleeding disorder. […] Prevalence: estimated to affect approx. 1% of the US population. […] Epidemiological data refers to the US, unless otherwise specified.

• #5 Von Willebrand Disease – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK459222/

  Von Willebrand disease is estimated to affect approximately 1% of the unselected population, but clinically significant disease prevalence is estimated to be about 125 per million, with severe disease affecting up to five per million. […] There is an equal distribution between males and females. Acquired von Willebrand disease prevalence is unknown but may represent 1% to 5% of all von Willebrand disease. Its prevalence is higher in certain groups. For example, it has been reported in up to 20% of malignancies and up to 100% of certain high flow states such as extracorporeal membrane oxygenation (ECMO) and metallic cardiac valves.

• #6 von Willebrand Disease: Practice Essentials, Etiology, Epidemiology

  https://emedicine.medscape.com/article/206996-overview

  Clinically significant vWD affects approximately 125 persons per million population, with severe disease affecting approximately 0.5-5 persons per million population. Reports from screenings of unselected individuals indicated a higher prevalence of vWD abnormalities, ie, close to 1% of the population. […] Males and females are affected equally by vWD. However, the phenotype may be more pronounced in females, because of menorrhagia and the greater visibility of bruises. […] In the great majority of cases, vWD is an inherited condition. Bleeding-related symptoms may occur at a young age, even just after or during birth. Some reports have suggested a decreased bleeding tendency as patients age.

• #7 Von Willebrand Disease (VWD) | Takeda U.S. Medical

  https://www.takedamedconnect.com/diseases-and-conditions/rare-hematology/von-willebrand

  Von Willebrand Disease (VWD), an inherited disorder characterized by a quantitative deficiency or qualitative defect of von Willebrand Factor (VWF), is the most prevalent inherited bleeding disorder.1,2,3 […] There are an estimated 23 to 110 cases of VWD per million people.5 Population-based estimates are higher, ranging from 0.6-1.3% of people. […] There are 3 types of VWD.2,6 Type 1 VWD, which accounts for ~75% of cases, is characterized by a reduced amount of VWF and with mild or moderate bleeding episodes. […] Type 2 VWD, which accounts for ~25% of cases, is characterized by qualitative defects of VWF that impair its function.2,6 This is associated with bleeding episodes that can range from mild to severe. […] Type 3 VWD accounts for 5% of cases, and is characterized by a near complete quantitative deficiency of VWF in plasma and with severe bleeding episodes.2,6

• #8 Orphanet: Von Willebrand disease type 1

  https://www.orpha.net/en/disease/detail/166078

  The type 1 disease is considered to be the most common form of VWD, accounting for between 50-75% of cases but its prevalence is probably overestimated. […] Prevalence: 1-5 / 10 000.

• #9 Von Willebrand Disease | Concise Medical Knowledge

  https://www.lecturio.com/concepts/von-willebrand-disease/

  Most common inherited bleeding disorder. […] Affects 1% of the general population. […] Equal prevalence between men and women, but women more likely to be symptomatic (as they experience menstrual bleeding). […] Inherited vWD: Type 1: 60%-80% of cases, Type 2: 20%-30% of cases, Type 3: 5% of cases. […] Acquired vWD: The true prevalence is not established. […] Has been described in 20% of patients with malignancies, with metallic cardiac valves, and on ECMO.

• #10 Orphanet: Von Willebrand disease

  https://www.orpha.net/en/disease/detail/903

  The prevalence of Von Willebrand disease (VWD) in the general population is estimated at between 0.6 and 1.3% (including all forms) depending on the study, but the prevalence of symptomatic VWD that requires specific treatment is approximately 1/10 000. Type 3 VWD is much more rare (1/1 000 000). […] VWD is most often transmitted in an autosomal dominant manner, however, the mode of inheritance is autosomal recessive for type 3 VWD and for some of the type 2 subtypes. Genetic counseling should be proposed to inform patients about the severity of the disease and the associated risks, and to allow screening for detection of other affected family members. […] For patients managed within specialized hemostasis hospital centers, the prognosis is favorable, even for those with the most severe forms of the disease.

• #11 Von Willebrand disease in Iran: diagnosis and management – Dorgalaleh – Annals of Blood

  https://aob.amegroups.org/article/view/4259/4991

  However, it seems that these data may not present the exact number of the patients with VWD in Iran and some patients may be misdiagnosed or remain undiagnosed due to the mild bleeding phenotypes and absence of well-equipped hemostasis laboratories around the country. […] Nevertheless, more than half of the registered patients with VWD are still not classified and therefore the exact prevalence of different types of VWD in Iran is unclear. […] In general, out of the 1,617 registered patients, 206 (12.7%) are listed as type 1, 199 (12.3%) as type 2, 304 (18.8%) as type 3, and 908 patients (56.1%) as unclassified. […] Overall, due to high rate of consanguineous marriage and autosomal recessive inheritance pattern of type 3, the prevalence of type 3 is around 4 per million in Iran, which overshadows that of Western European countries with a reported prevalence of ~0.45 per million.

• #12 von Willebrand Disease (vWD) Market | vWD Therapy and Key Companies

  https://www.delveinsight.com/blog/von-willebrand-disease-vwd-market

  Von Willebrand disease (VWD) is the most common bleeding disorder, affecting up to 1% of the population in the United States. This suggests that the disease affects 3.2 million people in the United States (about 1 in every 100). However, the general populations symptomatic von Willebrand disease prevalence is estimated to be between 23 and 110 per 1,000,000. As per DelveInsight estimates, the total diagnosed von Willebrand disease prevalence was 12,505 in the US in 2020, which is further expected to increase with a CAGR of 1.23% by 2030. Although von Willebrand disease occurs among men and women equally, women are more likely to notice the von Willebrand disease symptoms because of heavy or abnormal bleeding during their menstrual periods and after childbirth. The estimates show that females account for 55.50% of diagnosed VWD prevalent cases in the United States, with 6,941 cases in 2020.

• #13 Global Von Willebrand Disease Epidemiology Forecast to

  https://www.globenewswire.com/news-release/2020/02/17/1985636/0/en/Global-Von-Willebrand-Disease-Epidemiology-Forecast-to-2028.html

  The „Von Willebrand Disease (VWD) Epidemiology Forecast – 2028” report has been added to ResearchAndMarkets.com’s offering. This report delivers an in-depth understanding of the disease, historical, and forecasted epidemiology trends of Von Willebrand Disease (VWD) in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. […] According to this research, the total diagnosed prevalent population of Von Willebrand disease (VWD) in seven major markets was 33,758 in 2017. The number of Von Willebrand disease (VWD) cases in the 7MM is expected to increase during the study period of 2017-2028. Among the 7MM, the highest diagnosed prevalence of von Willebrand Disease (VWD) is estimated in the United States with 11,336 diagnosed cases in 2017. […] Comprehensive insight has been provided into the epidemiology Von Willebrand disease (VWD) in the 7MM countries covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. […] 7MM Total Diagnosed Prevalent Patient Population of von Willebrand Disease. […] Country Wise-Epidemiology of von Willebrand Disease.

• #14 Diagnosis and management of von Willebrand disease in Slovakia – Kubisz – Annals of Blood

  https://aob.amegroups.org/article/view/4284/html

  Von Willebrand disease (VWD) is a common bleeding disorder with prevalence in the Slovak Republic of 11.2 cases per 100,000 people. […] The referral-based prevalence per 100,000 inhabitants is 11.2 in Slovakia, a figure that is relatively high according to published referral-based prevalence around the world. […] The total Slovak population is 5,443,583 and the total number of patients with VWD in 2017 is 610 according to the National Registry of Congenital Bleeding Disorders in Slovakia, which is run by the National Haemophilia Centre, University Hospital and Medical School of Comenius University in Bratislava. […] The world-wide prevalence figures for type 3 VWD vary substantially as shown in Figure 2. It is surprising that Slovakia has one of the highest prevalence of VWD type 3 in the world. […] The prevalence of VWD in the Slovak Republic is approximately 11.2 cases per 100,000 people, but this is probably still underestimated due to the fact that many patients, particularly those with mild disease, continue to obtain care through primary care physician.

• #15 Von Willebrand disease epidemiology and demographics – wikidoc

  https://www.wikidoc.org/index.php/Von_Willebrand_disease_epidemiology_and_demographics

  The prevalence of von Willebrands disease is 0.6 to 1.3%. It is estimated that the referral prevalence of von Willebrands disease is approximately 1 case per 10,000 persons. […] The actual abnormality (which does not necessarily lead to disease) occurs in 0.9-3% of the population. […] The symptoms of VWD is disproportionately more common in women of child-bearing age. […] Although autosomal inheritance pattern of disease lead to an equal distribution of male patients and female patients, the disease has female predominance whose bleeding tendency shows during menstruation. […] There is no racial predilection to vWD however, it may be more severe or apparent in people with blood type O.

• #16 von Willebrand disease – Wikipedia

  https://en.wikipedia.org/wiki/Von_Willebrand_disease

  The prevalence of VWD is about one in 100 individuals. However, the majority of these people do not have symptoms. The prevalence of clinically significant cases is one per 10,000. Because most forms are rather mild, they are detected more often in women, whose bleeding tendency shows during menstruation. It may be more severe or apparent in people with blood type O.

• #17 Data and Statistics on von Willebrand Disease | Von Willebrand Disease (VWD) | CDC

  https://www.cdc.gov/von-willebrand/data/index.html

  Von Willebrand disease (VWD) occurs with equal frequency among men and women, affecting up to 1% of the general population. […] Women are more likely to experience symptoms of VWD because of the increased bleeding it causes during their menstrual periods, during pregnancy, and after childbirth. […] Between 2012 and 2023, more than 33,456 men, women, and children were seen at hemophilia treatment centers for treatment of VWD. About two thirds of them were women and girls.

• #18 Pediatric Von Willebrand Disease: Practice Essentials, Pathophysiology, Epidemiology

  https://emedicine.medscape.com/article/959825-overview

  von Willebrand disease is estimated to affect about 1% of the population. However, when considering symptomatic patients, an estimated 0.1% of the population is affected. […] Prevalence worldwide is estimated at 0.9-1.3%. […] The morbidity in individuals with von Willebrand disease varies. Many children with von Willebrand disease are asymptomatic. Some of these children have cutaneous and/or mucous membrane bleeding (eg, easy bruising, epistaxis). […] Menorrhagia is a common symptom in females with von Willebrand disease. It occurs in more than 50% of women with von Willebrand disease and may be the only clinical manifestation of the condition. […] A Swedish study, by Holm et al, found that compared with controls, patients with von Willebrand disease had a higher risk of hospitalization associated with cardiovascular disease (CVD; 1.3 fold) but a lower risk of CVD-linked mortality (0.4 fold). According to the investigators, this suggests that von Willebrand factor deficiency protects against arterial thrombosis even in the presence of atherosclerosis. […] von Willebrand disease affects males and females in equal numbers. […] von Willebrand disease is a congenital bleeding disorder and can be diagnosed at any age.

• #19 Von Willebrand Disease Epidemiology, Illness and Management | JBM

  https://www.dovepress.com/von-willebrand-disease-epidemiology-burden-of-illness-and-management-a-peer-reviewed-fulltext-article-JBM

  This systematic review also suggests that there are important gaps in VWD diagnosis that could delay care and result in adverse health outcomes. […] Patients with VWD had a high burden of bleeding events even after VWD diagnosis or if receiving VWD treatment. […] The reported real-world treatment patterns show a range of treatment options with respect to VWD types and severity. […] Findings from the identified sources also suggest that VWD is associated with poorer HRQoL scores as well as higher HCRU compared with general populations.

• #20 Von Willebrand Disease Epidemiology, Burden of Illness and Management: A Systematic Review

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9987238/

  Referral-based estimates were derived from the number of symptomatic patients attending hemophilia treatment centers or health care facilities based on national registries or surveillance studies. […] Population-based estimates relied on the identification of patients with VWD in cohort studies based on specific diagnostic criteria. […] Estimates of VWD prevalence also varied by disease type, age, and sex. […] This systematic review also suggests that there are important gaps in VWD diagnosis that could delay care and result in adverse health outcomes. […] The reported real-world treatment patterns show a range of treatment options with respect to VWD types and severity, with only a small proportion of patients with VWD reported to be receiving long-term VWF prophylaxis in a multinational VWD Prophylaxis Network study and in the Czech National Hemophilia Programme registry. […] This systematic review highlights the limited data available on the epidemiology of VWD, as well as the high disease burden and related impact on HRQoL and HCRU for patients with VWD.

• #21 von Willebrand Disease (vWD) Market | vWD Therapy and Key Companies

  https://www.delveinsight.com/blog/von-willebrand-disease-vwd-market

  Von Willebrand disease (VWD) is by far the most common type of bleeding condition, according to the World Federation of Hemophilia. It affects up to 1% of the world’s population. However, because most patients only have minor symptoms, only a tiny percentage of them know they have the disease. Patients with VWD are reported to be unaware of their disease in 9 out of 10 cases. Identifying VWD patients is a crucial challenge, and laboratory testing is difficult because no single test can provide a definitive diagnosis.

• #22 Paper: Primary Care Screening for Von Willebrand Disease in African American Adolescents with Heavy Menstrual Bleeding

  https://ash.confex.com/ash/2024/webprogram/Paper205652.html

  Von Willebrand Disease (VWD) is the most common inherited bleeding disorder; however, establishing a laboratory diagnosis can be challenging. […] In this analysis of an ongoing study examining the population prevalence of VWD in adolescents with heavy menstrual bleeding (HMB), we aim to compare VWF activity measured by RCo and GPIbM assay in African American participants with HMB and low RCo activity. […] In this small primary care population, we found that VWD is not uncommon in African American adolescents with HMB, occurring in 1 out of 20 participants who underwent testing. However, our data again demonstrate that African American individuals may have lower RCo activity, likely due to the D1472H polymorphism which could result in false labeling as VWD. Hematology consultation and further testing with GPIbM assay should be pursued for accurate diagnosis of VWD. VWF Ag may give some clues and in individuals with Ag 60% or higher but low RCo, a falsely low RCo should be considered, and confirmatory testing should be pursued.

• #23 Characteristics of Infants, Toddlers With von Willebrand Disease – Hematology Advisor

  https://www.hematologyadvisor.com/news/von-willebrand-disease-characteristics-infants-toddlers-risk/

  Diagnosing von Willebrand disease (VWD) in children younger than 2 is challenging, and data on this population is limited. […] The prospective study used data from the Centers for Disease Control and Prevention (CDC) Universal Data Collection (UDC) surveillance system established in the US Hemophilia Treatment Center Network (USHTCN). […] Overall, the study added more details to infants and toddlers with VWD, noting that family history plays an important role in diagnosis.

• #24

  https://haematologica.org/article/view/8278

  Molecular diagnosis of patients with von Willebrand disease is pending in most populations due to the complexity and high cost of conventional molecular analyses. The need for molecular and clinical characterization of von Willebrand disease in Spain prompted the creation of a multicenter project (PCM-EVW-ES) that resulted in the largest prospective cohort study of patients with all types of von Willebrand disease. […] Von Willebrand disease (VWD) is the most common bleeding disorder, with a reported incidence of 0.01% to 1%. […] Based on the results of a previous Spanish survey elucidating the difficulties of diagnosing VWD, a multicenter, prospective project (PCM-EVW-ES, Molecular and Clinical Profile of von Willebrand Disease in Spain) was designed to centrally characterize a large multicenter cohort of VWD patients, with inclusion of NGS molecular analysis.

• #25

  https://haematologica.org/article/view/8278

  The method presented herein has also been used in a smaller Portuguese cohort, with a similar diagnostic yield. […] Centralized comprehensive population studies, although logistically complex, would be the best way to obtain a clear picture of this disease and investigate connections between the multifactorial parameters that influence the diagnosis and etiology. […] In this study, 704 variants (237 unique) were compiled, whereas the total number of entries in EAHAD-VWD-LOVD is about 1,200 (708 unique), an indication of the effectiveness of this approach for the genetic study of large cohorts. […] The mutation detection rate was high compared with that of previous studies, being close to 100% in some VWD types. […] Accurate classification is particularly relevant for genetic counseling and detection of carriers in VWD types with autosomal recessive inheritance. […] The final obstacle to an accurate diagnosis is the uncertain pathogenicity of novel variations.

• #26 von Willebrand Disease Treatment & Symptoms Management | University of Utah Health | University of Utah Health

  https://healthcare.utah.edu/bleeding-clotting-disorders/von-willebrand-disease

  Von Willebrand disease (VWD) is a genetic bleeding disorder that is passed down from parent to child and affects both men and women. […] People with VWD do not make enough protein known as the von Willebrand factor (VWF). […] In general, people with mild to moderate VWD will experience minimal bleeding problems except when they have surgery or serious trauma. […] A person with severe VWD may have many of the same problems that a person with hemophilia has, including bleeding into joints. […] We will then conduct blood tests that are specific to different types of bleeding disorders. […] These tests will tell us if you have normal, low, or no blood clotting factor in your blood. […] Currently, there is no cure for von Willebrand disease. However, it is possible for changes in von Willebrand factor levels to occur with age, childbearing, and other factors that should be discussed with your hematologist.

• #27 von Willebrand Disease » Division of Hematology & Oncology » College of Medicine » University of Florida

  https://hemonc.pediatrics.med.ufl.edu/for-patients/hemophilia-and-hemostasis-treatment-center/von-willebrand-disease/

  Von Willebrand Disease is not as well known as hemophilia, but it is probably the most prevalent of all the disorders of bleeding. […] However, according to some estimates, it may affect as many as 1% of people in the United States. […] Today, we know it as a disease because those affected do not have enough functional von Willebrand factor. […] Specialized testing is required in order to complete the diagnosis of von Willebrand disease.

• #28 von Willebrand Disease: What It Is, Types, Symptoms & Treatment

  https://my.clevelandclinic.org/health/diseases/17709-von-willebrand-disease

  von Willebrand disease affects 1% of the U.S. population and is the most common bleeding disorder in the United States. Globally, von Willebrand disease affects an estimated 23 to 110 in 1 million people. […] Most people have von Willebrand disease because they inherited a mutated gene from one of their biological parents. This is autosomal dominant inheritance. Some people inherit mutated genes from both biological parents. This is autosomal recessive inheritance and is the most severe form of von Willebrand disease. […] Healthcare providers can treat von Willebrand disease but they cant cure it. Most people have Type 1 or Type 2 von Willebrand disease and may only need treatment if theyre injured or need surgery. People with Type 3 von Willebrand disease may need ongoing medical treatment to manage bleeding. […] Cleveland Clinic Childrens providers can accurately diagnose von Willebrand disease, a blood disorder. Well help your child manage symptoms and stay healthy.

• #29 Von Willebrand Disease (VWD) Market Clinical Trials, Unmet Needs and Forecast to 2032

  https://www.globaldata.com/store/report/von-willebrand-disease-market-analysis/

  The von Willebrand Disease (vWD) market size in the three major markets (3MM) (US, Germany, and UK) was $892 million in 2022. The vWD market will register a CAGR of more than 8% from 2022 to 2032. […] The report offers key topics covered including strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications for the vWD therapeutics market. […] The key countries in the vWD market are the US, UK, and Germany. In 2022, the US dominated the total vWD market in the 3MM occupying over 80% of the market share. The US’s market dominance is due to the higher price of pharmaceuticals in the US and the high diagnosed prevalence of vWD in the country compared to the other major markets. […] The vWD market is expected to have two novel entrants across the forecast period. These include vWF recombinant for pediatric use and BT-200, an RNA oligonucleotide targeting type 2 vWD. These novel therapies will provide significant opportunities to address the need for long-term, efficacious therapies.

• #30 von Willebrand disease is more common than what was previously thought | Research Communities by Springer Nature

  https://gengenocommunity.springernature.com/posts/von-willebrand-disease-is-more-common-than-what-was-previously-thought

  Von Willebrand disease (VWD) is a common bleeding disorder caused by mutations in the von Willebrand factor gene (VWF). The true global prevalence of VWD has not been accurately established. […] According to previous studies, VWD prevalence is estimated to vary between 0.6% and 1.3%, even though on the basis of cases referred to specialized centers about 1 case per 1,000 is estimated to have clinically relevant VWD. Despite this, the true prevalence of VWD has not been accurately established due to a lack of prospective and systematic studies and to the fact that some patients with VWF variants are asymptomatic or show mild clinical symptoms. […] Nowadays, a growing number of large-scale population-based sequencing studies are being conducted using next-generation sequencing (NGS). […] The high VWD prevalence established in this large-scale genetic epidemiology study indicates that the genetic predisposition to develop VWD due to VWF variants is likely to be more common than hitherto reported and also highlights that many patients carrying these variants are still not diagnosed. These data provide a hint that VWD is likely to be grossly underdiagnosed worldwide, which could contribute to undertreatment, significant (avoidable) morbidity, and health care system burden.

• #31 von Willebrand Disease (vWD) Market Opportunity Assessment, Epidemiology, Disease Management, Clinical Trials, Unmet Needs and Forecast to 2032

  https://www.marketresearch.com/GlobalData-v3648/von-Willebrand-Disease-vWD-Opportunity-35226641/

  The prevalence of vWD is increasing at a steady rate, in line with population growth in the 3MM, at an AGR of 0.61%. […] There are several unmet needs in this space, including the need for better diagnostic tools for vWD and improved therapies for severe vWD. […] Significant growth is expected throughout the forecast period as the vWD population steadily increases and novel therapies enter the market. […] Overview of vWD, including epidemiology, symptoms, diagnosis, and disease management. […] Annualized vWD therapeutics market revenue in 3MM (US, Germany and the UK), annual cost of therapy and treatment usage pattern in 2022 and forecast for 10 years to 2032; forecast covers three time points: base year, 5-year, and 10-year. […] Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the vWD therapeutics market. […] Pipeline analysis, comprehensive data assessing emerging trends and mechanisms of action under development for vWD treatment.

• #32 von Willebrand Disease (vWD) Market Opportunity Assessment, Epidemiology, Disease Management, Clinical Trials, Unmet Needs and Forecast to 2032

  https://www.researchandmarkets.com/reports/5892616/von-willebrand-disease-vwd-market-opportunity?srsltid=AfmBOop8UxoFgAxoV-7cbFYJc179xUXu07smZK1Q2P1f3T6-JhFwwfMp

  The prevalence of vWD is increasing at a steady rate, in line with population growth in the 3MM, at an AGR of 0.61%. […] Currently, the treatment regimen for vWD in the 3MM is mostly limited to DDAVP and coagulation factor VIII + vWF complex. […] There are several unmet needs in this space, including the need for better diagnostic tools for vWD and improved therapies for severe vWD. […] The vWD late-stage RD pipeline is focused on developing vWF recombinant for pediatric use, RNA oligonucleotide therapy BT-200. […] Significant growth is expected throughout the forecast period as the vWD population steadily increases and novel therapies enter the market. […] Overview of vWD, including epidemiology, symptoms, diagnosis, and disease management. […] Annualized vWD therapeutics market revenue in 3MM (US, Germany and the UK), annual cost of therapy and treatment usage pattern in 2022 and forecast for 10 years to 2032; forecast covers three time points: base year, 5-year, and 10-year.

• #33 von Willebrand Disease (vWD) Market Opportunity Assessment, Epidemiology, Disease Management, Clinical Trials, Unmet Needs and Forecast to 2032

  https://www.giiresearch.com/report/gd1360024-von-willebrand-disease-vwd-market-opportunity.html

  The prevalence of vWD is increasing at a steady rate, in line with population growth in the 3MM, at an AGR of 0.61%. […] Overview of vWD, including epidemiology, symptoms, diagnosis, and disease management. […] Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the vWD therapeutics market. […] Analysis of the current and future market competition in the 3MM vWD therapeutics market. Insightful review of the key industry drivers, restraints, and challenges. Each trend is independently researched to provide a qualitative analysis of its implications.

• #34 Von Willebrand disease: MedlinePlus GeneticsLock

  https://medlineplus.gov/genetics/condition/von-willebrand-disease/

  Von Willebrand disease is estimated to affect 1 in 100 to 10,000 individuals. Because people with mild signs and symptoms are not likely to seek medical attention, this condition may be underdiagnosed. Most researchers agree that von Willebrand disease is the most common genetic bleeding disorder. […] Von Willebrand disease can have different inheritance patterns. Most cases of type 1 and type 2 von Willebrand disease are inherited in an autosomal dominant pattern, which means one copy of the altered gene in each cell is sufficient to cause the disorder. Type 3, some cases of type 2, and a small number of type 1 cases of von Willebrand disease are inherited in an autosomal recessive pattern, which means both copies of the gene in each cell must have a variant to cause the disorder. The parents of an individual with an autosomal recessive condition each carry one copy of the altered gene, but they typically do not show signs and symptoms of the condition.

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