Articles tagged with Hemophilia
Researchers from the University of Miami Miller School of Medicine and Sylvester Comprehensive Cancer Center will present their work on various hematological conditions at ASH 2025. These posters highlight recent findings in fields such as von Willebrand disease, multiple myeloma, and acute myeloid leukemia.
A new survey found that 72% of respondents agree that plasma-derived medicines can save lives, but most haven't donated. Plasma is essential in producing treatments for various serious health conditions.
Researchers designed long-acting human albumin-fused FIX variants with unique pharmacokinetic properties, including extended plasma half-lives and enhanced extravascular distribution. The findings endorse the use of engineered albumin-fused FIX variants as personalized therapy options for hemophilia B.
Researchers at Cincinnati Children's Hospital Medical Center have successfully grown liver tissue that can produce its own internal blood vessels. This breakthrough could lead to new treatments for people living with hemophilia and those experiencing acute or chronic liver failure, as the liver organoids can secrete coagulation factors.
A recent study has discovered that saliva contains special vesicles with extrinsic tenase complexes, which trigger rapid coagulation of the blood in haemophilic patients. This finding provides significant contributions to understanding the disease and its treatment options.
A clinical trial demonstrated the efficacy and tolerability of concizumab, a new drug for haemophilia patients, reducing bleeding rates. Concizumab works by blocking an anticoagulant factor, restoring the delicate balance between clotting and anti-clotting factors.
A single-dose gene therapy has been shown to significantly reduce bleeding episodes in adults with hemophilia B, with an average reduction of 71% compared to standard treatment. The therapy, which enables the liver to produce clotting factor IX, has been FDA-approved for use in patients with this genetic disorder.
Researchers at the University of Hawaii have developed a new gene editing technology that can efficiently deliver healthy genes to the body. This method addresses limitations of current methods and has shown success rates of up to 96%, potentially leading to faster and more affordable treatments for various genetic diseases.
A phase 3 randomised controlled trial found monthly injections of fitusiran effective in reducing bleeds in patients with severe haemophilia A or B. The study included 56 male patients with inhibitors, who experienced no bleeds after nine months on prophylactic treatment.
A global study involving McMaster University researchers has trialled a potential new treatment for hemophilia A, a life-threatening genetic blood disorder. Once-weekly injections of efanesoctocog alfa can prevent bleeding and promote near-normal activity by the clotting agent factor VIII.
Wilate prophylaxis showed an 84% reduction in bleeding episodes and improved quality of life in children and adults with all types of VWD. The treatment reduced the frequency of bleeding episodes by lowering the mean total annualized bleeding rate compared to on-demand treatment.
A study by Sanford Burnham Prebys found that misfolded proteins in liver cells contribute to liver cancer development, potentially increasing the risk of certain patients receiving gene therapies for hemophilia. Improving FVIII folding could decrease this risk and develop safer treatments.
A study by the University of Bonn elucidated an important immune mechanism crucial for making hemophilia treatment effective. The researchers found that regulatory T cells can activate a self-destruct button on B cells against factor VIII, leading to its tolerance.
A new type of adeno-associated virus (AAV) gene therapy candidate, FLT180a, has been shown to reduce bleeding risk in patients with haemophilia B. The treatment led to sustained production of FIX protein from the liver in nine out of ten patients, eliminating the need for regular replacement therapy.
A novel gene therapy has shown sustained expression of clotting factor VIII, leading to a reduction or complete elimination of bleeding events in patients with hemophilia A. The trial demonstrated improved production of coagulation factor VIII over prolonged periods.
A new pouch device has been developed to protect transplanted human liver cells from immune systems for up to six months, producing crucial biomolecules. This breakthrough offers a potential path toward treating human diseases without needing to suppress the patient's immune system.
Researchers at the University at Buffalo have developed a new treatment that pairs essential proteins with lysophosphatidylserine to build immune tolerance and prevent antibody development, potentially treating autoimmune disorders and allergies.
A recent study found that amniotic fluid-derived, neonatal, and adult cells can be used to deliver long-lasting Factor VIII protein for Hemophilia A treatment. The researchers identified cells from umbilical cord tissue as the most promising candidates, which yielded high levels of Factor VIII mRNA and blood clotting activity.
A study by Indiana University School of Medicine researchers has found that B cell activating factor (BAFF) plays a role in the generation and maintenance of inhibitors in hemophilia A patients. Blocking BAFF with a combination therapy may induce tolerance in these patients, offering a potential solution to treatment failure.
A CHOP-led research study found that B cell activating factor (BAFF) levels are elevated in patients resistant to coagulation protein therapy, suggesting a potential target for new treatments. Anti-BAFF therapies combined with immune tolerance therapies may tame the immune response in some patients with severe hemophilia A.
The American Society of Hematology, International Society on Thrombosis and Haemostasis, National Hemophilia Foundation, and World Federation of Hemophilia have developed joint clinical practice guidelines for von Willebrand Disease (VWD). The guidelines provide recommendations for diagnosis and management, including the use of injecta...
Two new posters will present investigative treatment options using NUWIQ and WILATE for rare bleeding disorders. The research focuses on long-term prophylaxis with Simoctocog Alfa for hemophilia A, as well as the use of a von Willebrand Factor/Coagulation Factor VIII Complex for treatment of inherited platelet disorders.
Two large-scale genetic studies identified over 7,000 regions of the human genome influencing blood cell characteristics. These findings bring us closer to using genetic scoring in clinics to predict personal risk of developing blood disorders.
Researchers at Wake Forest Institute for Regenerative Medicine have developed an optimized cellular platform for delivering Factor 8 to treat patients with hemophilia A. The new approach uses human placental cells to produce therapeutic levels of Factor 8, potentially providing a long-term correction for the disease.
Children with severe hemophilia A who receive early prophylaxis are less likely to have joint damage in young adulthood than those who begin treatment later. However, prophylaxis does not fully prevent joint damage.
A breakthrough gene therapy treatment has been shown to offer long-term benefits for people with Haemophilia A, transforming the lives of 13 UK patients. The treatment resulted in a substantial fall in bleeding rates three years after treatment, with none requiring regular factor VIII injections.
Women who started estrogen therapy before age 60 with their ovaries removed experienced reduced mortality over long-term follow-up. However, older women or those above 70 without ovaries did not derive this benefit and may have adverse effects of treatment.
A new study by McMaster University researchers reveals that over 1,125,000 men globally have hemophilia, with 418,000 having severe forms of the disease. The study found a significant life expectancy disadvantage for those with hemophilia, particularly in lower-income countries.
Researchers found threshold levels of circulating factor VIIa that prevented bleeding episodes in animal models, potentially leading to a new preventive treatment for patients with hemophilia who develop antibodies to standard maintenance therapy.
A new protein called emicizumab, administered via subcutaneous injection, has shown significant reductions in bleeding episodes in patients with haemophilia A. Weekly and fortnightly dosing regimens have been found to reduce bleed rates by 96% and 97%, respectively.
RCSI and Bayer are working together to create new, tailored treatments for people with severe haemophilia. The goal is to promote blood clotting with increased precision, improving the quality of life for those affected by this condition.
Hemophilia B can be treated for life with a single injection containing disease-free liver cells that produce the missing clotting factor. The finding uses stem-cell strategies and CRISPR/Cas9 gene editing to repair mutations in the patient's FIX gene, allowing normal blood clotting.
A groundbreaking gene therapy trial has shown that over one year on from a single treatment, participants with haemophilia A are showing normal levels of the previously missing protein, effectively curing them. The results have particular significance as the first successful gene therapy trial for the haemophilia A.
Researchers report remarkable benefits from new therapies for bleeding and clotting disorders, including emicizumab's high effectiveness in preventing bleeds in children with hemophilia A. Additionally, a gene therapy treatment provides durable improvement in clotting factor levels and long-term protection from bleeds.
The Phase 1/2 clinical trial showed that SPK-9001 reduced annualized bleeding rate by 97% and factor IX concentrate use by 99% in participants with hemophilia B. The data suggest a one-time infusion of SPK-9001 may sustain factor IX activity levels, reducing bleeding episodes.
Researchers found a shift in cytokine profiles of mice with FVIII inhibitor development, including increased levels of pro-inflammatory cytokines. A negative correlation was also observed between risk factors for FVIII inhibitor development and age at gene therapy administration.
A new gene therapy has successfully produced sustained levels of clotting factor in adult hemophilia B patients, eliminating the need for regular infusions. The treatment, which uses a highly active variant of the clotting factor, achieved significant clinical benefits with no adverse effects.
A novel monoclonal antibody called emicizumab has been shown to decrease the incidence of bleeding episodes in patients with hemophilia A and inhibitors by 87%. This breakthrough therapy offers a more effective and safer alternative to traditional treatments, reducing the need for frequent dosing and minimizing side effects.
Researchers develop plant-based therapy to prevent antibodies from forming against clotting factor VIII, a common cause of hemophilia reactions. The treatment showed promising results in dog trials, with significantly lower antibody levels and no negative side effects.
Researchers reported the highest and most sustained levels of clotting factor IX in hemophilia B patients after a single dose of experimental gene therapy. Patients were able to discontinue factor infusions and engage in normal activities without disabling bleeding episodes, significantly improving their quality of life.
Researchers at the University of Pennsylvania have developed a dual gene therapy approach using CRISPR/Cas9 to deliver key components to treat hemophilia B. The study showed stable Factor IX activity in newborn and adult mice, with all treated mice surviving a partial liver removal without complications.
Researchers have developed a biodegradable capsule that can administer protein therapy to treat hemophilia B, providing hope for a more accessible and affordable treatment. The oral delivery system contains micro- and nanoparticles that carry the protein therapy and are designed to release it over time, alleviating the burden of inject...
Researchers develop a new therapy that prolongs thrombin production, potentially treating both hemophilia A and B, including those with antibodies against standard therapy. The treatment could also accelerate clot formation in patients with factor V Leiden, offering a more effective and long-acting alternative to current therapies.
Blocking furin protein may offer unexpected benefits for patients with hemophilia A. Gene therapy and protein replacement treatments may be more effective with this approach.
Researchers examined documents on direct-to-consumer marketing of hemophilia treatment products, revealing a strategy that begins in childhood and continues into adulthood. The marketing strategies focus on personal relationships and foster brand loyalty, with companies spending millions to target consumers.
A new study published in the New England Journal of Medicine found that patients with severe hemophilia A who received human plasma-based treatments developed inhibitors at a lower rate than those who received recombinant Factor VIII. This could lead to more effective treatment options for families affected by this rare genetic condition.
A new study analyzing 50 years of hemophilia care data found significant disparities in outcomes between men with severe and mild forms of the disease. Men with severe hemophilia were more likely to experience physical limitations, disability, and frequent bleeding despite access to effective therapies. The study emphasizes the need fo...
A new antibody called ACE910 has shown promising results in reducing the need for frequent injections in patients with severe hemophilia A. The study found that ACE910 is safe and effective in preventing excessive bleeding, even in patients who develop antibodies to existing treatments.
A team from Penn Dental Medicine successfully produced a shelf-stable, low-cost drug using freeze-dried lettuce leaves, which promotes tolerance to clotting factors for hemophilia patients. The study confirms the viability of plant-based biopharmaceutical production on a commercial scale.
Outpatient pharmacy expenditures totaled $475,718,130 for publicly insured children with serious chronic illness in California. Antihemophilic factor was the most expensive medication, accounting for 98% of its class's expenditures and 41% of total pharmacy expenditures.
Dr. Qizhen Shi's team aims to investigate a novel gene therapy approach that induces immune tolerance for hemophilia A, overcoming the challenge of inhibitory antibodies. The project seeks to develop a clinically translatable gene therapy protocol that provides therapeutic FVIII protein.
Researchers at Children's Hospital Los Angeles found that a new recombinant factor VIII fusion protein prolongs the duration of anti-hemophilia factor in children. This allows for reduced frequency of treatment, reducing the burden on patients with severe hemophilia A.
Researchers have successfully treated dogs with hemophilia using a mutant human protein with unusually high blood-clotting power. The treatment eliminated pre-existing antibodies and reversed the bleeding disorder in two years without causing thrombosis or immune reactions.
Katherine High, Amit Nathwani, Arthur Nienhuis, and Andrew Davidoff honored for their groundbreaking research on gene therapy for hemophilia B. They developed successful clinical applications using adeno-associated virus vectors and demonstrated stable therapeutic expression of the clotting factor.
Researchers at Stanford Medicine have devised a new way to edit the genome without using enzymes or promoters, which may lead to a safer and longer-lasting treatment for genetic diseases like hemophilia. The technique successfully cured mice with hemophilia by inserting a gene for a clotting factor missing in the animals.
Researchers from Penn and Florida developed a plant-based approach to prevent antibodies forming against clotting factor proteins in people with hemophilia. This method, which uses genetically modified plants to induce tolerance, showed promise in preventing inhibitor formation and even reversing it in mice.
A new study uses genetically modified plants to teach the immune system to tolerate clotting factors, reducing the risk of antibody formation. The treatment, which involves feeding mice with plant capsules, has shown promising results and may offer a cost-effective alternative to current treatments.
Dr. Katherine High's groundbreaking research in gene therapy has led to novel approaches to correcting hemophilia, with ongoing studies in a current NIH-funded clinical trial. The E. Donnall Thomas Prize recognizes her remarkable contributions to the field, transforming the notion of using genetically engineered mechanisms for treatment.
A new gene therapy has led to a dramatic decline in bleeding events in dogs with naturally occurring hemophilia A. The treatment involves packaging specialized blood platelets with genes that express clotting factor, resulting in significantly fewer serious bleeding events over three years.
Researchers found that people with hemophilia infected with HIV for decades have increased levels of immune cells targeting HIV. This cellular response, combined with antiretroviral therapy, enables patients to long-term survive despite HIV activity. The study provides new insights into the reasons behind clinical stability in these pa...