factor VII

First participant with Von Willebrand Disease dosed with HMB-002, a novel subcutaneous therapy designed to increase endogenous von Willebrand Factor and Factor VIII levels COPENHAGEN, Denmark and CAMBRIDGE, MA, USA I February 27, 2025 I Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced the first participant with Von Willebrand Disease (VWD) has been dosed in Velora Pioneer, a Phase 1/2 clinical trial investigating HMB-002, a potential first-in-class subcutaneous therapy for VWD. “We are excited to advance the clinical development of HMB-002 for Von Willebrand Disease,” said Catherine Rea, MD, PhD, Vice President of Clinical Development at Hemab. “HMB-002 is the first fixed-dose subcutaneously administered antibody-based preventative therapy aiming to transform daily life for patients.” HMB-002 is a monovalent antibody uniquely designed to increase levels of von Willebrand Factor (VWF) and Factor VIII and provide a long-acting, subcutaneous prophylactic treatment for individuals with all types of VWD. Preclinical data for this investigational therapy were first presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), showcasing its potential to target the underlying patho-etiology of VWD by stabilizing VWF and promoting sustained hemostatic correction. “Von Willebrand Disease is characterized by a deficiency in VWF activity and sometimes associated with lowered levels of FVIII – both essential to control severe bleeding,” said Dr. Priyanka Raheja, the Royal London Hospital, Barts Health NHS Trust. “HMB-002 functions by increasing VWF and FVIII and could offer a complete disease correction for some and substantial modification for bleeding tendency for others. I am excited to be a contributing investigator on the Phase 1/2 trial.” The Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in individuals with VWD. The initial part of the study will evaluate single ascending doses of HMB-002 in a controlled setting. The first participant was dosed at Richmond Pharmacology in London under the supervision of Principal Investigator, Dr. Ulrike Lorch. The study is actively enrolling participants, with interim data anticipated later this year. “The initiation of the HMB-002 clinical program establishes Hemab as a multi-asset clinical-stage biotech and underscores our unwavering commitment to reimagine blood clotting therapies for people living with a variety of underserved bleeding disorders,” said Benny Sorensen, MD, PhD, CEO of Hemab. “Von Willebrand Disease has been documented for about 100 years. However, the current standard of care is sparse, ancient, and inconvenient. It’s time to leapfrog treatment into the 21 st century, and we believe HMB-002 can become a new back-bone therapy for all types of Von Willebrand Disease.” In addition to Velora Pioneer, Hemab is also conducting Velora Discover , a prospective natural history study collecting data on bleeds, treatments, and quality-of-life elements in people with VWD. Hemab is partnered with Haemnet, a specialist research and communications consultancy in the bleeding disorders community, to conduct VWD 360, the largest-ever natural history study in all types of VWD based on people’s detailed reported lived experience. Recent initial results were shared at EAHAD 2025. About Von Willebrand Disease Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, characterized by quantitative or qualitative defects in Von Willebrand Factor (VWF), often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding in women. The severity of bleeding ranges from low-volume events to potentially life-threatening hemorrhages. Chronic blood loss frequently leads to iron deficiency anemia, exacerbating the disease burden and reducing quality of life, particularly for those with clinically understated subtypes. Despite its prevalence, current treatment options for VWD primarily focus on managing symptoms rather than addressing the underlying defect in VWF production or function. About HMB-002 HMB-002 is a monovalent human antibody developed as the first-in-class prophylactic treatment for Von Willebrand Disease targeting the underlying root cause of the disease, a condition driven by a deficiency or defect in Von Willebrand Factor (VWF), a key regulator of hemostasis. By specifically targeting the C-terminal CK domain of VWF, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function. Preclinical data suggest strong potential for meaningful therapeutic benefit. For more information, please visit clinicaltrials.gov (NCT06610201). About Hemab Therapeutics Hemab is a multiple clinical-asset biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA, and Copenhagen, Denmark, Hemab is progressing a pipeline of innovative therapeutic solutions, leveraging a variety of cutting-edge technologies and approaches to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5 TM , targets building a pipeline of multiple development programs to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com . Follow us on LinkedIn , Facebook , Instagram , and X . SOURCE: Hemab Therapeutics

Interim results demonstrate treatment with HMB-001 resulted in >50% reduction in treated bleeds at all tested dose levels In second oral presentation, Hemab presented first preclinical data for HMB-002, a potential novel treatment for Von Willebrand Disease Results from lived experience of Glanzmann thrombasthenia (GT360) and Von Willebrand Disease (VWD 360) natural history studies were included in two additional oral presentations COPENHAGEN, Denmark and CAMBRIDGE, Mass., Feb. 7, 2025 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today presented interim data from the ongoing evaluation of HMB-001, a novel bispecific antibody in development as first ever prophylactic treatment for the bleeding disorder Glanzmann thrombasthenia (GT). The Phase 2 study consists of a minimum 6-week prospective run-in where participants record bleeds via an electronic bleed diary, followed by 3 months of treatment with HMB-001. Interim efficacy data to date demonstrated >50% reduction in treated bleeds in all 3 tested dose cohorts. Underscoring its potential to address unmet medical needs and to expedite patient access, HMB-001 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration and the UK Medicines and Healthcare products Regulatory Agency has awarded it designation under the Innovative Licensing and Access Pathway. Hemab also unveiled compelling preclinical data for HMB-002, a potential novel treatment for Von Willebrand Disease (VWD). These data were featured as oral presentations at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) held this week in Milan, Italy. "Glanzmann thrombasthenia is a serious and devastating bleeding disorder where patients often experience in excess of 60-80 treatment-requiring bleeds annually. These interim Phase 2 results signal the potential of a new reality for the Glanzmann thrombasthenia community, bringing us a step closer to a much-needed breakthrough in preventing bleeding," said Kate Madigan, Chief Medical Officer at Hemab. "The preclinical data for HMB-002 highlights its potential to specifically address the underlying patho-etiology and revolutionize care for the Von Willebrand Disease community, offering hope to prevent life-threatening complications and provide patients with a critical new lifeline." Interim pharmacokinetic (PK) data from the Phase 1/2 study of HMB-001 supports the use of weekly or biweekly dosing regimens. Pharmacodynamic (PD) responses are aligned with the mechanism of action, demonstrating a dose-dependent accumulation of activated Factor VII (FVIIa) and enhanced potentiation through TLT-1 binding. Efficacy assessments indicate a reduction of over 50% in treated bleeding episodes at all tested dose levels. In the single ascending dose (SAD) study, doses of 0.2, 0.5, and 1.25 mg/kg were evaluated, while the multiple ascending dose (MAD) study explored biweekly doses of 0.3, 0.6, and 0.9 mg/kg. The findings suggest a safe and effective dosing range of 0.3-0.6 mg/kg, while doses of 0.9 mg/kg and above were associated with elevated D-dimer levels and risk of venous thrombosis. These results provide valuable insights into optimizing dose and regimen for improved outcomes. "Bleeding disorders such as Glanzmann thrombasthenia present significant hardships, including unpredictable and life-altering bleeds, physical pain, social stigma, and emotional isolation. Despite these burdens, preventive treatments have yet to be developed," said Suthesh Sivapalaratnam, MD, PhD, Consultant Hematologist at Barts Health NHS Trust and Associate Professor at Queen Mary University of London. He adds: "With a bleed reduction of greater than 50%, the Phase 2 results highlight the transformative potential of HMB-001, offering people with Glanzmann thrombasthenia and providers a long-awaited breakthrough that could redefine care standards and bring renewed hope to a community in urgent need of innovation." "As a principal investigator in this study, I've seen firsthand how HMB-001 has the potential to significantly improve outcomes for people with Glanzmann thrombasthenia," said Laurent Frenzel, MD, PhD, Head of the Hemophilia Treatment and Research Center at the Necker-Enfants Malades Hospital (Paris Cité). "The Phase 2 data demonstrate promising efficacy and a manageable safety profile, representing a significant advancement in the treatment landscape. I look forward to seeing these results translate into a meaningful impact for those who have long faced limited options and unmet medical needs." Hemab plans to complete Phase 2 study recruitment in the first half of 2025. Hemab presented compelling preclinical data for HMB-002, a monovalent antibody uniquely designed to address VWD by increasing endogenous Von Willebrand Factor (VWF) levels and improving overall hemostasis. Unlike current treatments that often require frequent intravenous infusions or focus on symptom management, HMB-002 has the potential to provide a long-lasting, subcutaneous prophylactic solution that targets the root cause of VWD. Data demonstrate a promising safety and efficacy profile with high-affinity target-selective binding and preservation of key physiological functions of VWF when HMB-002 is bound. Administration of HMB-002 to cynomolgus monkeys resulted in a time-dependent accumulation of VWF, a corresponding rise in VWF activity, and a rise in the amount of another important blood clotting protein called Factor VIII (FVIII). The distribution of different sizes of VWF molecules (multimers) remained largely the same, suggesting that the activity levels of VWF were consistent. These results indicate that HMB-002, by boosting the levels of naturally occurring VWF, has the potential to be an effective, convenient and long-lasting preventive treatment option for people with VWD. The company is actively enrolling participants in VELORA-Discover, a prospective observational study for individuals with Type 1 VWD, to further understand the disease's natural history and support future clinical trials. Hemab's partner Haemnet, a specialist research and communications consultancy in the bleeding disorders community, also presented results from two natural history studies. The Glanzmann's 360 and Von Willebrand Disease 360 studies showcased the unmet needs and burdens the patients face every day. About Glanzmann Thrombasthenia Glanzmann thrombasthenia (GT) is a rare and severe bleeding disorder marked by debilitating, sometimes life-threatening bleeding episodes. Results from an international Glanzmann's 360 (GT360) natural history study revealed the substantial burden of this disease: 88% of the 117 participants reported at least one bleed in the previous week, with 34% of those bleeds requiring medical treatment. These bleeding episodes significantly impact patients' mental health and quality of life, with 67% reporting low mood, 52% reporting emotional problems, and 46% experiencing social isolation. Additionally, 81% of participants reported missing school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for GT. About HMB-001 HMB-001 is a subcutaneously administered bispecific antibody that binds and stabilizes endogenous Factor VIIa with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for the accumulation of endogenous Factor VIIa in the body, recruitment of Factor VIIa directly to the surface of the activated platelets, where it is known to facilitate hemostatic plug formation. HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann thrombasthenia (GT) with the potential to treat other debilitating rare bleeding disorders. The U.S. Food and Drug Administration granted Fast Track Designation and Orphan Drug Designation to HMB-001 for the treatment of GT while the UK The Medicines and Healthcare products Regulatory Agency has awarded it designation under the Innovative Licensing and Access Pathway (ILAP). For more information, please visit clinicaltrials.gov (NCT06211634). About Von Willebrand Disease Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, characterized by quantitative or qualitative defects in Von Willebrand Factor (VWF), often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding in women. Severity of bleeding ranges from low-volume events to potentially life-threatening hemorrhages. Chronic blood loss frequently leads to iron deficiency anemia, exacerbating the disease burden and reducing quality of life, particularly for those with clinically understated subtypes. Despite its prevalence, current treatment options for VWD primarily focus on managing symptoms rather than addressing the underlying defect in VWF production or function. About HMB-002 HMB-002 is a monovalent human antibody developed as the first-in-class prophylactic treatment for Von Willebrand Disease targeting the underlying root cause of the disease, a condition driven by a deficiency or defect in Von Willebrand Factor (VWF), a key regulator of hemostasis. By specifically targeting the C-terminal CK domain of VWF, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function. Preclinical data suggest strong potential for meaningful therapeutic benefit. For more information, please visit clinicaltrials.gov (NCT06610201). About Hemab Therapeutics Hemab is a clinical-stage biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA, and Copenhagen, Denmark, Hemab is progressing a pipeline of innovative therapeutic solutions, leveraging a variety of cutting-edge technologies and approaches to transform the treatment paradigm for patients with high unmet need. The company's strategic guidance, Hemab 1-2-5TM, targets building a pipeline of multiple development programs to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com. Follow us on LinkedIn, Facebook, and X. Media Contact: Peg Rusconi [email protected] 617-910-6217 SOURCE Hemab Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Haemophilia is a disorder in which the blood does not clot properly. There are two main forms: haemophilia A, and the rarer haemophilia B. The two types of haemophilia have different precise causes but the same symptoms.1 Haemophilia affects males much more commonly, and usually more severely, than females.2,3 The condition is characterised by slow and inefficient formation of blood clots resulting in prolonged bleeding and spontaneous internal bleeds. When a healthy individual sustains an injury – internal or external – which causes bleeding, the injury (trauma) triggers a process known as the coagulation cascade, also sometimes called the clotting cascade. This cascade is a series of reactions in the bloodstream whose end result is haemostasis – the stopping of a bleed. In fact, the cascade starts as two series of steps which originate separately. These are known as the intrinsic pathway and the extrinsic pathway. These two pathways converge into a third pathway, called the common pathway. The mechanism by which blood coagulates involves a series of clotting factors circulating in the bloodstream, each activating the next in a specific order.4 If any part of this intricate process fails, there is a possibility that the blood will not clot properly and the individual will bleed excessively. In haemophilia, one of the clotting factor proteins is partly or completely missing. People with haemophilia take longer than normal for bleeding to stop after an injury. They may also have bleeding into joints and muscles without having had an injury.5 • Haemophilia A is a deficiency of factor VIII (8). • Haemophilia B (also known as Christmas Disease) is a deficiency of factor IX (9).6 Other rare bleeding disorders exist: Haemophilia C, also known as plasma thromboplastin antecedent (PTA) deficiency or Rosenthal syndrome. This is a mild form of haemophilia affecting both sexes and is caused by factor XI deficiency.7 It affects about 1 person per million,8 far fewer people than haemophilia A or B. Von Willebrand Disease, a genetic bleeding disorder where patients have a deficient or defective von Willebrand factor (VWF), a protein in the blood that is important for clot formation.9 Very rarely, individuals may be born with, or develop: Factor I Deficiency, Factor II Deficiency, Factor V Deficiency, Factor VII Deficiency, Factor X Deficiency, Factor XII Deficiency, or Factor XIII Deficiency.10 Immune thrombocytopenia (ITP), a rare autoimmune disease affecting platelet production. ITP is different from haemophilia, although some of the symptoms are similar. People with ITP have reduced platelets but the rest of their clotting mechanism works normally.11,12 Haemophilia A affects about 1 in 5,000 of the male population. Haemophilia B is rarer, affecting around 1 in 30,000 of the male population. Severe forms of haemophilia are very rare in women. Around 30 to 50 per cent of females who are carriers may have milder forms of haemophilia. Haemophilia affects people of all ethnic origins and from all parts of the world.14 The symptoms of haemophilia A and B are the same: Bruises Bleeding into muscles and joints, which may cause: o Aching o Swelling o Pain and stiffness o Difficulty using a joint or muscle Spontaneous bleeding (sudden internal bleeding for no apparent reason, including life-threatening bleeds i.e. intracranial) Prolonged bleeding after surgery or having a tooth extracted Prolonged bleeding after an accident, especially after an injury to the head16 If an individual is showing some or all of the symptoms of haemophilia, a formal diagnosis may be recommended, especially if there is a family history of haemophilia.17,18 Initially the patient may be invited to take a general blood test, or screening, for blood clotting. If haemophilia is suspected, the next step might be referral for specific tests at a haemophilia treatment centre for factor VIII and factor IX. Haemophilia is classed as mild, moderate or severe depending based on the baseline level of clotting factor a person has, as measured in a laboratory test. The same measures are used for haemophilia A and B. Percentage of normal factor activity in blood 50-150% Number of international units (IU) per millilitre of blood 0.5-1.5 IU/mL Percentage of normal factor activity in blood 5-40% Number of international units (IU) per millilitre of blood 0.05-0.4 IU/mL When bleeds typically occur Bleeding following injury or surgery Percentage of normal factor activity in blood 1-5% Number of international units (IU) per millilitre of blood 1.01-0.05 IU/mL When bleeds typically occur Bleeding as a result of minor injury Percentage of normal factor activity in blood < 1% Number of international units (IU) per millilitre of blood < 0.01 IU/mL When bleeds typically occur Spontaneous bleeding into joints and muscles Haemophilia occurs in response to an alteration, or mutation, in the gene responsible for producing clotting factor VIII or IX, resulting in the factor not being adequately produced. In haemophilia A, the gene responsible for producing factor VIII, known as the F8 gene, is altered. In haemophilia B, the gene responsible for producing factor IX, known as the F9 gene, is altered. Haemophilia is usually an inherited condition, passed on from parents to offspring. However, in about a third of cases of haemophilia A and B there is no family history of the condition. This is because inherited haemophilia can arise as a spontaneous germline mutation – that is, when there is an alteration in DNA in offspring which is not present in the parental DNA but which can then be passed on to the next generation.22,23 This type of gene alteration is also known as a de novo mutation.24,25 Victoria, Queen of the United Kingdom from 1837 to 1901, was a carrier of haemophilia B. The mutation is believed to have arisen spontaneously. Victoria's youngest son, Leopold, was affected by the disease and at least two of her five daughters, Alice and Beatrice, were carriers. In rare cases, a person can develop haemophilia at some stage in life even when there is no family history and no spontaneous mutation. This is known as acquired haemophilia (AH). AH is an autoimmune condition – where the body mistakenly attacks healthy cells or tissue. In AH, the body produces antibodies (known as inhibitors) that attack clotting factors, most commonly factor VIII. It affects mainly older people and is believed to affect approximately one person in a million.26 Haemophilia A and B are recessive sex-linked, or X-linked, mutations. To understand what this means, a basic understanding of genetics is needed. In the human body, every cell normally contains 23 pairs of chromosomes. These chromosome pairs carry all of our genetic material. Each chromosome contains hundreds to thousands of genes. Genes are what carry the instructions for making proteins. Twenty-two of our chromosome pairs, called autosomes, look the same in both males and females. The 23rd pair, the sex chromosome pair, is different in males and females. Females have two copies of the X chromosome (XX), while males have one X and one Y chromosome (XY).27,28 The F8 gene and the F9 gene (responsible for producing factors VIII and IX respectively) happen to be located on the X chromosome, which means that they affect males and females in different ways. If a female has a recessive mutation for haemophilia (A or B) on only one of her X chromosomes, she will be a carrier. This means that she will herself be unaffected by the disease, or may have it in a mild form, but she can pass the mutation on to her progeny. On average half of her sons will be affected and half of her daughters will be carriers. If a male has the same mutation on his X chromosome, he will be affected by the disease because he doesn’t have another X chromosome that is free from the mutation. He will pass the altered gene on to any daughters via the X chromosome that he contributes. He cannot pass it on to his sons, because he contributes only the Y chromosome to them. Many years ago, hospital-based blood transfusions were among the few treatments available for people with haemophilia and the life expectancy of a person with severe haemophilia was low. A good deal of progress has been made in recent decades and years. Contemporary treatments have revolutionised the landscape for individuals with haemophilia, empowering them to surpass conventional expectations and engage more extensively in life. Modern therapies not only enable more active participation in physical pursuits, including sports, but also can enable people with haemophilia to carry out routine activities free from the constant concern of potential bleeding episodes. For children, this progress translates into the opportunity to join their peers in different activities and fosters a more inclusive life experience. Plasma DerivedPlasma-derived factor concentrates are derived from human plasma. These products are purified from donated blood plasma, providing a natural source of clotting factors. Rigorous screening and purification processes are employed to minimise the risk of transmitting infections. Plasma derived treatments factors have a standard half-life.29 RecombinantRecombinant factor concentrates are produced through genetic engineering techniques, creating clotting factor proteins in a laboratory setting. This method helps ensure a consistent and pure product, reducing the risk of contamination and adverse reactions. Recombinant concentrates can include standard, extended or high-sustained half-life factors.30 Standard Half-Life Factor (SHL)Standard half-life factor concentrates involve injections of clotting factor with a traditional half-life, requiring regular administrations to maintain adequate levels in the bloodstream.31 This form of treatment can be used in on-demand therapy and prophylaxis.32 Extended Half-Life Factor (EHL)Extended half-life factor concentrates are designed to remain active in the bloodstream for a longer duration, allowing for higher factor levels with less frequent injections. This approach aims to improve bleed control and reduce the frequency of treatment, especially in prophylactic regimens33,34,35, 36. Non-Factor TherapyNon-factor therapies in represent treatment approaches that target the underlying haemostatic defect without directly replacing clotting factors like factor VIII or IX. They aim to enhance haemostasis by either mimicking factor function or rebalancing the coagulation system, offering alternatives to traditional factor replacement therapy. All non-factor therapies to date are administered subcutaneously.35 Gene TherapyGene therapy involves introducing a functional copy of the clotting factor gene into the patient's cells, enabling the body to produce the deficient clotting factor. This approach aims to provide a long-term correction of the underlying genetic cause of haemophilia.36 For most people, treatment works extremely well. However, for some, their bodies react as if the clotting factor were a foreign substance. Their immune system then develops antibodies known as inhibitors, which means that the injected clotting factor may not work as well or work at all. The antibodies may disappear spontaneously after a period of regular treatment, or a higher dose of the injection may be needed for a period of time. In some people, the antibodies remain and their bleeding must be controlled using a different treatment.37 Liberate Life is Sobi’s vision for haemophilia and our way of supporting and empowering people living with the condition. Liberate Life is based on the fundamental criteria that treatment should always allow people living with haemophilia to feel safe, be protected from bleeds, have long-term joint health protection, and not feel burdened by haemophilia. The Liberate Life website aims to give people with haemophilia support and information that may help to identify personal goals, limit the impact of the condition, and get the most out of care and treatment. More than 75 per cent of people with haemophilia around the world have limited or no access to diagnosis and treatment, particularly in the developing world. Together with Sanofi, we intend to donate up to 1 billion international units (IUs) of factor replacement for use in developing countries. Learn more about our commitment to the WFH Humanitarian Aid Program here: Humanitarian aid 1Understanding Haemophilia, The Haemophilia Society https://haemophilia.org.uk/wp-content/uploads/2017/04/Understanding_haemophilia_WEB.pdf 2Genetic causes of haemophilia in women and girls. Table 1. National Library of Medicine. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8132474/ 3Haemophilia. Better Health Channel. https://www.betterhealth.vic.gov.au/health/conditionsandtreatments/haemophilia 4Chaudhry, R.; Usama, S.M.; Babiker, H.M. Physiology, Coagulation Pathways. National Library of Medicine. https://www.ncbi.nlm.nih.gov/books/NBK482253/ 5Haemophilia. The Haemophilia Society. https://haemophilia.org.uk/bleeding-disorders/haemophilia-a-and-b/ 6Haemophilia, The Haemophilia Society. https://haemophilia.org.uk/bleeding-disorders/haemophilia-a-and-b/ 7Factor XI Deficiency. Medscape. https://emedicine.medscape.com/article/209984-overview 8Thejus, J. et al. Hemophilia C: A Case Report With Updates on Diagnosis and Management of a Rare Bleeding Disorder. National Library of Medicine. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7153668/ 9What is von Willebrand Disease (VWD)? bleedingdisorders.com. https://www.bleedingdisorders.com/about/von-willebrand-disease 10Other rare bleeding disorders. bleedingdisorders.com. https://www.bleedingdisorders.com/about/types-of-bleeding-disorders 11Hemophilia, Differential Diagnosis. Rare Disease Advisor. https://www.rarediseaseadvisor.com/disease-info-pages/hemophilia-differential-diagnosis/ 12Difference Between Thrombocytopenia and Hemophilia. Difference Between.com. https://www.differencebetween.com/difference-between-thrombocytopenia-and-vs-hemophilia/ 13Understanding Haemophilia, The Haemophilia Society. https://haemophilia.org.uk/wp-content/uploads/2017/04/Understanding_haemophilia_WEB.pdf #3 14Understanding Haemophilia, The Haemophilia Society. https://haemophilia.org.uk/wp-content/uploads/2017/04/Understanding_haemophilia_WEB.pdf #3 15E-Learning. World Federation of Haemophilia. https://elearning.wfh.org/elearning-centres/hemophilia/ 16E-Learning. World Federation of Haemophilia. https://elearning.wfh.org/elearning-centres/hemophilia/ 17Hemophilia. Medline Plus. https://medlineplus.gov/hemophilia.html 18Hemophilia. Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/14083-hemophilia 19Understanding Haemophilia, The Haemophilia Society. https://haemophilia.org.uk/wp-content/uploads/2017/04/Understanding_haemophilia_WEB.pdf #2 20About Haemophilia. NHS Southern Haemophilia Network. https://www.southernhaemophilianetwork.org/about/haemophilia/what-is/ 21How Hemophilia is Inherited. CDC. Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/hemophilia/inheritance-pattern.html 22Hemophilia A, National Hemophilia Foundation. https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-a 23Hemophilia B, National Hemophilia Foundation. https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-b 24Hemophilia A. NORD. https://rarediseases.org/rare-diseases/hemophilia-a/ 25Haemophilia B. ScienceDirect. https://www.sciencedirect.com/topics/biochemistry-genetics-and-molecular-biology/haemophilia-b 26Acquired Hemophilia. NORD. https://rarediseases.org/rare-diseases/acquired-hemophilia/ 27How many chromosomes do people have? Medline Plus. https://medlineplus.gov/genetics/understanding/basics/howmanychromosomes/ 28Human Genome Project FAQ, NIH. https://www.genome.gov/human-genome-project/Completion-FAQ 29Treatment of Haemophilia. Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/hemophilia/treatment.html 30Treatment of Haemophilia. Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/hemophilia/treatment.html 31Haemophilia Treatment. Irish Haemophilia Society. https://haemophilia.ie/about-bleeding-disorders/haemophilia/haemophilia-treatment-2/ 32SHL/EHL Fact Sheet. World Federation of Hemophilia. https://www1.wfh.org/publications/files/pdf-2365.pdf 33Berntorp E, Negrier C, Gozzi P, Blaas PM, Lethagen S. Dosing regimens, FVIII levels and estimated haemostatic protection with special focus on rFVIIIFc. Haemophilia. 2016;22(3):389-396. 34Current Treatments. National Bleeding Disorders Foundation. https://www.hemophilia.org/bleeding-disorders-a-z/treatment/current-treatments 35Shapiro et al. Real-world data demonstrate improved bleed control and extended dosing intervals for patients with haemophilia B after switching to recombinant factor IX Fc fusion protein (rFIXFc) for up to 5 years. Haemophilia 2020 Nov;26(6):975-983 36Tagliaferri A, Matichecchia A, Rivolta GF, Riccardi F, Quintavalle G, Benegiamo A, Rossi R, Coppola A. Optimising prophylaxis outcomes and costs in haemophilia patients switching to recombinant FVIII-Fc: a single-centre real-world experience. Blood Transfus. 2020;18(5):374-385 37Current Treatments. National Bleeding Disorders Foundation. https://www.hemophilia.org/bleeding-disorders-a-z/treatment/current-treatments 38Inhibitors. The Haemophilia Society. https://haemophilia.org.uk/bleeding-disorders/inhibitors/