Current Hemophilia News and Events

Current Hemophilia News and Events, Hemophilia News Articles.
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Organizations collaborate to develop international von Willebrand Disease guidelines
The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), and World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on the diagnosis and management of von Willebrand Disease (VWD), the world's most common inherited bleeding disorder. The guidelines were published today in Blood Advances. (2021-01-12)

Octapharma USA presents bleeding disorders research at virtual ASH annual meeting
Octapharma USA will present its most recent rare bleeding disorders research initiatives during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, a virtual medical congress to be held December 5 - 8. (2020-12-01)

Tiny engineered therapeutic delivery system safely solves genetic problems in mice
Inserting genetic material into the body to treat diseases caused by gene mutations can work, scientists say - but getting those materials to the right place safely is tricky. Scientists report in the journal Science Advances that the lipid-based nanoparticles they engineered, carrying two sets of protein-making instructions, showed in animal studies that they have the potential to function as therapies for two genetic disorders. (2020-08-21)

A new cell & gene therapy approach to treat common bleeding disorder
WFIRM researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A. (2020-07-28)

Early treatment linked to reduced joint damage in young adults with severe hemophilia A
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, according to a new study in Blood Advances. While data suggest earlier initiation of prophylaxis provides some protection compared to later initiation of prophylaxis, prophylaxis does not fully prevent joint damage. (2020-06-03)

Scientists find way to supercharge protein production
Researchers at Washington University School of Medicine have found a way to increase production of proteins in bacteria up to a thousandfold, a discovery that could aid production of proteins used in the medical, food, agriculture, chemical and other industries. (2019-12-18)

Hemophilia three times more prevalent than thought
For the World Federation of Hemophilia, Iorio assembled an international team of researchers from France, US and UK to perform a meta-analysis of the registry data in countries with the most comprehensive registries of hemophilia, which were Australia, Canada, France, Italy, New Zealand and the UK. (2019-09-09)

Researchers ready B cells for novel cell therapy
Scientists at Seattle Children's Research Institute are paving the way to use gene-edited B cells -- a type of white blood cell in the immune system -- to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first-of-its-kind in clinical trials at Seattle Children's in as soon as five years. (2019-05-02)

Gene-based factor VIIa prevents bleeding episodes in animals with hemophilia
Hematology researchers have further refined how a treatment currently used on an urgent basis to control bleeding in hemophilia patients holds promise as a preventive treatment as well. A study in animals may set the stage for a new therapy for a subset of patients with hemophilia who now develop antibodies to the standard maintenance treatment and then require on-demand 'bypass' therapy. (2019-04-15)

Tissue model reveals how RNA will act on the liver
MIT researchers have shown an engineered model of human liver tissue can be used to investigate nucleic acid-based therapies, such as RNA interference, before testing them in patients. (2019-03-05)

Genetic relic of the 'black death' may offer clues in treating liver disease
A gene mutation that is believed to have safeguarded some people in 14th century Europe from the bubonic plague today may be protecting HIV patients co-infected with hepatitis C from potentially fatal liver scarring, says a University of Cincinnati (UC) College of Medicine physician-scientist. (2018-12-03)

Red glow helps identify nanoparticles for delivering RNA therapies
A new screening process could dramatically accelerate the identification of nanoparticles suitable for delivering therapeutic RNA into living cells. The technique would allow researchers to screen hundreds of nanoparticles at a time, identifying the organs in which they accumulate - and verifying that they can successfully deliver an RNA cargo into living cells. (2018-10-01)

Electron microscopy provides new view of tiny virus with therapeutic potential
Researchers from the Salk Institute and the University of Florida are reporting how they used cryo-EM to show the structure of a version of a virus called an AAV2, advancing the technique's capabilities and the virus' potential as a delivery vehicle for gene therapies. (2018-09-07)

New results support continued development of AAV-based human factor VIII gene therapy
Gene therapy using an optimized adeno-associated virus (AAV) to deliver the human fac-tor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expres-sion and no detectable antibody response for 30 weeks in some animals. (2018-08-20)

New platform discovered at City of Hope poised to be next generation of genetic medicines
A City of Hope scientist discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the next generation of genetic therapies. This editing platform may be used to cure inherited and acquired diseases. The proof-of-concept study spotlights a promising new gene-editing platform that may eventually be used to treat diseases such as sickle cell disease, hemophilia and other genetic disorders. (2018-07-16)

Mechanical force controls the speed of protein synthesis
As cells create proteins, the proteins modulate synthesis speed by exerting a mechanical force on the molecular machine that makes them, according to a team of scientists who used a combination of computational and experimental techniques to understand this force. (2018-05-16)

Single injection treats hemophilia B for life, in proof-of-concept study
Salk researchers have demonstrated in mice that hemophilia B can be treated for life with one single injection containing disease-free liver cells that can produce their missing clotting factor. (2018-05-01)

Breakthrough brings gene-editing medicine one step closer to patient applications
Researchers at the University of Alberta have discovered a way to greatly improve the accuracy of gene-editing technology by replacing the natural guide molecule it uses with a synthetic one called a bridged nucleic acid, or BNA. The research promises to bring the technology much closer to therapeutic reality. (2018-04-13)

Look! Down in the petri dish! It's a superplatelet!
A University of British Columbia bioengineer has endowed platelets with extra powers to make the clotting process more resilient in the face of trauma. If it's proven to work in clinical situations, such 'superplatelets' might become a standard part of emergency department supplies, along with bandages, oxygen and saline. (2018-04-12)

Study explores emerging role of NAD+ in innate and adaptive immune responses
Researchers at Brigham and Women's Hospital (BWH) have discovered a new cellular and molecular pathway that regulates CD4+ T cell response -- a finding that may lead to new ways to treat diseases that result from alterations in these cells. (2018-02-23)

Researchers find adult endothelial stem cells that can make fully functional blood vessels
Stem cells are increasingly used to treat disorders caused by defective tissues. The repair of blood vessels by vascular endothelial stem cells (VESCs) is an attractive therapeutic option, but the existence of this type of stem cell has not been conclusively shown. Researchers identified a VESC that can regenerate blood vessels and treat a rare bleeding disorder. These VESCs have the potential to serve as a new cell-based therapy for blood vessel-related diseases. (2018-02-15)

Global hematology diagnostics market estimated to expand at a robust CAGR over 2021
Hematology includes various IVD technologies such as blood analysis, flow cytometry, immunodiagnostics, molecular diagnostics, hemostasis, histology, and cytology. (2018-02-08)

Self-sealing miniature 'wound' created by engineers
Biomedical engineers have developed a miniature self-sealing model system for studying bleeding and the clotting of wounds. The researchers envision the device as a drug discovery platform and potential diagnostic tool. (2018-02-07)

New therapies improve outlook for bleeding and clotting disorders
In three studies being presented today during the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, researchers report remarkable benefits from new, more easily administered therapies for bleeding and clotting disorders, including hemophilia A and people with cancer experiencing venous thromboembolism. (2017-12-11)

Spark Therapeutics and Pfizer announce publication in The New England Journal of Medicine of Interim Data from phase 1/2 clinical trial of investigational gene therapy for Hemophilia B
Spark Therapeutics (NASDAQ: ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer Inc. (NYSE:PFE), today announced that The New England Journal of Medicine has published interim data as of July 25, 2017, from the Phase 1/2 clinical trial of SPK-9001, an investigational gene therapy for hemophilia B. (2017-12-07)

Inflammatory factors linked to inhibition of factor VIII gene therapy in hemophilia A
As a gene therapy cure for hemophilia A moves closer to reality, a new study sheds light on a challenging complication in which a host autoimmune response inhibits the production of normal clotting factor VIII from the transferred FVIII gene. (2017-12-07)

Spark Therapeutics announces publication in The Lancet of pivotal Phase 3 clinical trial data for investigational voretigene neparvovec
Spark Therapeutics (NASDAQ: ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced The Lancet, a highly ranked peer-reviewed journal, has published Phase 3 clinical trial data of voretigene neparvovec, an investigational, potential one-time gene therapy candidate for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). (2017-07-14)

Disruptive technology for the treatment of hemophilia
An international team of hematologists including Guy Young, MD, of Children's Hospital Los Angeles, has found that in patients with hemophilia A with inhibitors, a novel therapy called emicizumab, decreases incidence of bleeding episodes by 87 percent. Results of this multicenter phase III study called HAVEN 1, will be presented at the International Society of Thrombosis and Hemostasis and published in the New England Journal of Medicine on July 10. (2017-07-10)

Altered virus may expand patient recruitment in human gene therapy trials
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response. (2017-06-13)

Scripps Florida scientist wins $2 million grant to study childhood disorder
Assistant Professor Seth Tomchik of the Florida campus of The Scripps Research Institute (TSRI) has received $2 million in funding from the National Institutes of Health's National Institute of Neurological Disorders and Stroke (NINDS). The new five-year grant funding will support the study of neurofibromatosis type I, an inherited disorder that results from genetic mutations affecting a protein called neurofibromin (Nf1). (2017-05-31)

Researchers improve vbectors for delivering hFVIII gene therapy to treat Hemophilia A
A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to take forward into development. (2017-03-31)

Don't kill the messenger RNA
Success of new protein-making therapy for hemophilia opens door for treating many other diseases. (2017-02-14)

Plant-made hemophilia therapy shows promise, Penn study finds
Researchers from the University of Pennsylvania School of Dental Medicine and University of Florida have developed a therapy to prevent a significant complication of hemophilia treatment. Results in dogs show promise. (2017-02-13)

Overcoming hurdles in CRISPR gene editing to improve treatment
The new gene-editing tool CRISPR/Cas9 holds promise for new treatment of such genetic diseases as cystic fibrosis, muscular dystrophy and hemophilia. But to work well, it must be delivered across the cell membrane and into its nucleus, a process that can trigger cell defenses and 'trap' CRISPR/Cas9, reducing its treatment potential. Now, Vincent Rotello's laboratory at UMass Amherst has designed a delivery system using nanoparticles to assist CRISPR/Cas9 across the membrane and avoid entrapment by cellular machinery. (2017-02-07)

Scripps Florida team awarded $1.8 million grant to develop drugs for heart disease and rheumatoid ar
Scientists from the Florida campus of The Scripps Research Institute (TSRI) have been awarded approximately $1.8 million from the National Institute of General Medical Sciences of the National Institutes of Health to develop a series of drug candidates for a number of diseases, including heart disease, rheumatoid arthritis and several neurodegenerative disorders. (2017-01-24)

Katherine High talks gene therapy progress for hemophilia & inherited retinopathies
Gene therapy has shown some of its most promising early results in treating patients with hemophilia and inherited retinal disorders that cause vision loss and blindness, both important research and drug development targets during the career of Katherine High, M.D., President and Chief Scientific Officer of Spark Therapeutics. (2017-01-04)

Gene therapy for liver disease advancing with the help of adeno-associated viral vectors
Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing. The potential for continued technological improvements to expand the therapeutic applications of gene therapy to treat liver disorders and the remaining clinical challenges are examined in a comprehensive review article published in Human Gene Therapy. (2016-12-30)

After gene therapy, hemophilia B patients maintain near-normal levels of clotting factor
Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing them to stop clotting factor infusions and to pursue normal activities of daily life without disabling bleeding episodes. (2016-12-03)

Penn scientists use CRISPR for first time to correct clotting in newborn and adult mice
CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. For the first time, researchers have developed a dual gene therapy approach to deliver key components of a CRISPR/Cas9-mediated gene targeting system to mice to treat hemophilia B. (2016-11-30)

UT Austin engineers develop first-ever capsule to treat hemophilia
Thanks to a breakthrough led by researchers in the Cockrell School of Engineering at the University of Texas at Austin, treatment for hemophilia can now be administered via a biodegradable system, a capsule, giving people affected by the hereditary bleeding disorder hope for a less expensive, less painful treatment option than conventional injections or infusions. (2016-11-28)

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