Articles tagged with Hemophilia
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Katherine A. High, MD, receives the 2013 E. Donnall Thomas Lecture and Prize for her pioneering research on gene therapy for hemophilia and inherited retinal degenerative diseases. Her work has led to sustained clinical improvements using adeno-associated virus vectors.
After over 20 years, a team led by Professor Merlin Crossley has found the genetic puzzle piece for hemophilia B Leyden, a rare disorder that improves symptoms after puberty. The breakthrough demonstrates the importance of persistence and curiosity in scientific research.
Researchers discovered that thrombin activity propagates through blood in steady waves, crucial for sealing large wounds but potentially leading to complications. This finding sheds light on the mechanisms behind severe bleeding in hemophilia patients and has implications for other cardiovascular disorders.
A study found that vigorous physical activity increases the risk of bleeding in children with moderate or severe hemophilia A or B. Most bleeds associated with physical activity occur within an hour of activity, and the absolute increase in risk may be small.
A new treatment for hemophilia A has shown significant reductions in bleeding events and infusion frequency, particularly in children and adolescents. The study suggests that prophylaxis is the optimal treatment for patients with severe hemophilia, improving quality of life.
A recent clinical trial in patients with hemophilia B showed that Factor IX gene therapy was able to convert severe hemophilia to moderate or mild disease. The treatment demonstrated a sustained therapeutic effect and eliminated or substantially reduced the need for standard protein replacement.
Researchers have combined gene therapy and stem cell transplantation to successfully reverse hemophilia A in large animals, eliminating symptoms and restoring normal activity. However, the treatment's effects may be reduced or shorter due to an immune response developed by the treated animals.
A new treatment for severe hemophilia A patients with inhibitors has been shown to be highly effective in preventing bleeding events. The study, published in the New England Journal of Medicine, found that FEIBA infused three times a week resulted in a 62% reduction in all bleeding events and a 72% reduction in target joint bleeding.
Researchers have developed a genetically engineered clotting factor that controls bleeding in animal models of hemophilia. The variant protein is safer and more effective than existing treatments, with potential to treat a range of bleeding disorders.
The researchers are using genetically modified plants to encapsulate a tolerance-inducing protein within plant cells to prevent fatal allergic reactions in patients with hemophilia A and B. The new funding will help propel the research to determine if the technique can work in other models and potentially to clinical trials.
Vanessa Oliveira, Portuguese researcher, develops strategy to reprogramme immune system of hemophilia patients. Her project aims to improve treatment efficacy by inducing patient's immune tolerance towards therapeutic clotting factors.
Researchers used genome editing to treat hemophilia in mice by precisely targeting and repairing mutated DNA. The treatment, which uses zinc finger nucleases, showed clinically meaningful results with no toxic effects or complications.
A commentary highlights the shortcomings of Canada's current drug surveillance system, citing the off-label use of recombinant activated factor 7 (rFVIIa) as a case in point. The authors emphasize the need for improved collaboration between payers, insurers, and manufacturers to ensure safe monitoring of high-risk drugs.
A systematic review of recombinant activated factor 7 (rFVIIa) use in patients without hemophilia found uncertain benefits and potential risks. The review suggests that the drug's use should be restricted to clinical trials due to its limited effectiveness.
Pfizer's pre-clinical study on recombinant factor Xa therapy and engineered recombinant factor VIIa molecule shows promising results in restoring hemostasis. The company is committed to advancing hemophilia care through its pipeline and research initiatives.
A new plant-based system has been developed to help hemophilia patients build tolerance to their treatment, reducing the risk of allergic reactions. The system, made from genetically modified plants, can be ingested and releases a tolerated protein into the small intestine, where it can be acted on by the immune system.
Research highlights the need to address hemophilia treatment disparities in developing countries. A new study from North Carolina State University found that prevalence of hemophilia A has increased over time due to improved treatments, but access to life-saving clotting factor VIII remains severely limited.
Dr. Keri Smith has received a Career Development Award from the National Hemophilia Foundation to develop a therapy for people with Hemophilia A. Her approach involves altering chemical signals produced by T cells that trigger immune responses, making individuals more receptive to replacement Factor VIII.
Researchers at the University of Minnesota Medical School have successfully provided long-term expression of Factor VIII in hemophilia A mice using a new gene therapy approach, marking a promising step toward human clinical trials.
Researchers develop gene therapy approach to target Factor VIII in hemophilia A mice, reducing disease symptoms. L-Asparaginase degradation mechanism also uncovered, with potential for optimizing treatment.
The World Federation of Hemophilia launches a video podcast on comprehensive care, emphasizing the need for multidisciplinary teams to provide accurate diagnosis, prompt treatment, and support for families. Comprehensive care is crucial in both developing and developed countries to improve patient outcomes and prevent complications.
Researchers have redesigned factor VIII to increase its ability to drive blood clotting, which could lead to more effective and less burdensome hemophilia treatment. The new design improves the stability of the protein, allowing it to withstand manufacturing processes and exposure to the human bloodstream.
Researchers have developed a chemically modified protein that neutralizes inhibitor antibodies in people with Hemophilia A, offering a potential new treatment option. The modified protein, called electrophilic FVIII analog (E-FVIII), permanently inactivates these antibodies, paving the way for more effective blood clotting correction.
The NHLBI has published its first US clinical practice guidelines for von Willebrand Disease, a condition that affects 1 in 100 to 1,000 people. The guidelines provide recommendations for screening, diagnosis, and treatment of the three major types of VWD, emphasizing proper diagnosis and treatment to prevent complications.
Researchers at Einstein College of Medicine successfully cured hemophilia A in an animal model by transplanting healthy liver endothelial cells from donor mice. The study overturned conventional wisdom regarding the source of factor VIII, a crucial clotting protein lacking in people with type A hemophilia.
A study on 776 patients with angioedema without urticaria reported a practical approach to differential diagnosis. The European approach to diagnosis and management of angioedema is encouraged for its effectiveness.
A novel gene therapy could provide a permanent genetic cure for hemophilia A patients, including those with expensive and ineffective treatments. The treatment involves cloaking Factor VIII in platelets to avoid antibody attacks, making it effective even in patients with inhibitory antibodies.
Researchers replaced a faulty gene responsible for hemophilia with a transposon from fish, preventing profuse bleeding in neonatal mice. The approach holds promise for treating the most common form of hemophilia, affecting nearly 18,000 Americans.
Hematologists say coagulation-factor concentrates from plasma are now deemed safe from most infectious agents, but challenges from new agents remain. Recombinant products offer a risk-free alternative, but safety studies have not definitively made the case for either.
Researchers at the University of Iowa developed a new gene therapy vector that can effectively deliver therapeutic genes to liver cells, converting Hemophilia A from a severe to a mild form in mice. The correction lasted 30 weeks and showed promise for reducing bleeding episodes in people with hemophilia.
Researchers have discovered a promising new treatment option for haemophilia and heart disease by targeting the P-selectin protein. Infusion of P-selectin could provide an affordable and effective means of achieving clotting to stop bleeding incidents in haemophiliacs.
Researchers have achieved a cure for haemophilia in dogs using gene transfer strategies. The study identified key problems to overcome before applying this approach in humans, including mode of delivery.
Researchers at the University of Rochester Medical Center have designed a new version of factor VIII that doubles its ability to bind with factor IX, potentially reducing costs and immune reactions. The redesigned protein accelerates blood clotting, offering a promising alternative for next-generation hemophilia treatment.
A breakthrough in gene therapy has successfully corrected hemophilia in laboratory animals by producing a normal level of clotting factor activity and preventing bleeding incidents.
Researchers at UNC School of Medicine used embryonic stem cells treated with a growth factor to reverse hemophilia B in genetically altered mice. The study demonstrates the potential for stem cell therapy in treating genetic disorders, such as liver diseases.
A study by Frits Rosendaal and colleagues found that haemophilia carriers have a significantly lower risk of ischaemic heart disease, with a 36% decrease in overall mortality. This reduction in blood clotting may offer protection against heart attacks and has implications for future prevention strategies.
A new gene therapy tool has shown promising results in treating mice with hemophilia A by correcting the faulty factor VIII gene. The technology uses RNA trans-splicing to produce normal factor VIII protein, offering a potential cure for this genetic disorder.
Dr. Katherine High, a leading gene therapy researcher, has been chosen by the Howard Hughes Medical Institute for her groundbreaking work on treating hemophilia B. Her research holds promise for delivering therapeutic genes directly into patients' cells to improve blood clotting and quality of life.
The BAXJECT device eliminates filter needles during Factor VIII concentrate infusion preparation, reducing risk of needle-stick injury and contamination. The device is designed for safe and easy use by children and adults alike.
Baxter's rAHF/PFM therapy has shown bioequivalence with Recombinate, a current gold-standard, indicating comparable hemostatic efficacy in managing Haemophilia A. This new method reduces theoretical risk of disease transmission by eliminating human or animal-derived raw materials.
Gene therapy has shown promising results in hemophilia trials, with patients experiencing significant improvements in quality of life. The treatment involves inserting a normal version of the defective gene to produce clotting factor, and has been tested in small animals before being moved to human trials.
A study found that 35% of staff left the field within four years due to colleague stress. However, burnout was not a major factor; instead, support from professional colleagues significantly impacted employee retention. The researchers conclude that improving team relationships can reduce job stress and enhance retention.
Researchers have developed a gene therapy using human blood outgrowth endothelial cells to treat hemophilia A. The treatment, which has shown long-term attainment of robust levels of factor VIII, is promising and may pave the way for testing in humans.
A clinical study found that children with severe hemophilia have a high risk of developing deep venous thrombosis linked to long-term catheter implants. Eight out of 15 boys in the study developed blood clots in the veins near their hearts.
Dusty Miller, PhD, received the National Hemophilia Foundation's 1999 Dr. Murray Thelin Researcher of the Year Award for his work on gene therapy, a potential treatment for hemophilia. His pioneering research has led to the development of retroviral gene-transfer kits and promising early-stage gene-therapy trials.
Researchers report encouraging early results in a novel gene therapy designed to improve the clinical course of hemophilia B, with two patients requiring significantly less infused clotting factor to treat symptoms. No adverse side effects were experienced by any of the patients treated.
New gene therapy study in mice demonstrates a gradual increase in factor VIII levels, stable for 11 months. The goal is one dose, which could be all that's needed to be physiologically cured of future bleeds.
Researchers at UPMC have successfully conducted the world's first in vivo gene therapy for hemophilia A using a modified retrovirus to deliver the FVIII gene. The treatment aims to permanently restore clotting factor production, reducing costs and complications associated with the disease.
Researchers have successfully used gene therapy to partially correct protein deficiency responsible for hemophilia B in dogs, suggesting its potential for human treatment. The corrected genes produced blood clotting protein factor IX steadily more than a year and a half after treatment.