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UniQure recently announced plans to “expeditiously advance” its investigational hemophilia B gene therapy AMT-061 into a pivotal study in 2018. The announcement followed meetings the company had with both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency.
Researchers have made an important discovery that could change the way doctors understand and treat inhibitor development in hemophilia A
Actions from the President adds uncertainty to the health insurnace market.
Halloween can be even more meaningful this year by promoting an altruistic lifestyle and giving back to your community. Check out these 13 spooky Halloween fundraising ideas.
The National Hemophilia Foundation is pleased to announce that Michael Kuklish of Lillian, Alabama, is the recipient of the 2017 Kevin Child Scholarship.
Focusing on positive thoughts can actually impact your overall health. How, you ask? Today we dive in and see how positive thoughts can make you healthier.
This activity discusses the latest advances in the management of hemophilia.
October happens to be Adopt a Shelter Dog Month. So, how can you celebrate this month and support another incredible cause? We have some cool canine concepts.
San Francisco-based Catalyst Biosciences, Inc. recently announced positive clinical data from the first group of patients participating in an ongoing Phase 1/2 proof-of-concept study in individuals with severe hemophilia B. The trial, which is being conducted at three centers in South Korea, is designed to evaluate Catalyst’s investigative, subcutaneous factor IX (FIX) therapy known as CB 2679d. Catalyst develops its therapies using engineered proteases, enzymes that increase the rate of chemical reactions within cells.
It can be difficult to decide how to give, what you can give or even where to give to. Today let's take a closer look at how to donate with a purpose to disaster relief.
The findings showed the value of collaboration between HTCs, insurers, and speciality pharmacies.
The mission of the Patient Reported Outcomes, Burdens, and Experiences (PROBE) study is to investigate and directly probe patient perspectives on outcomes they deem relevant to their life and care. PROBE aims to develop a new global tool to enhance the direct patient-voice in health care decision-making. The study has partners in both developed and developing countries.
Members of the National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council (MASAC) recently gathered during NHF’s 69th Annual Meeting on August 26, 2017 in Chicago. The meeting generated three new documents related to treatment, therapies, women with bleeding disorders, carriers and emergency room management. These documents, which are listed below, were subsequently adopted by NHF’s Board of Directors on September 17, 2017.
Call your Senator and ask them to vote NO on the Graham-Cassidy bill, which would jeopardize access to healthcare for people with bleeding disorders.
The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “Bacteremia: The Good, The Bad, and The Ugly.”
Art galleries are a beneficial component of any neighborhood. Check out some of the ways art galleries are important and why you should visit them this fall.
September 9, 2017 saw the passing of Harold R. Roberts, MD at the age of 87. Over the course of his more than 50-year career, the North Carolina native was a highly regarded physician, teacher, researcher, author and mentor in the field of hematology.
Researchers at the Boston Children’s Program in Cellular and Molecular Medicine and the Harvard Medical School recently made important discoveries relevant to the functioning of von Willebrand factor (VWF). The study, “Flow-induced Elongation of von Willebrand Factor Precedes Tension-Dependent Activation,” was published online, August 23, 2017, in the journal Nature Communications.
Turns out, regular writing and keeping a journal actually has a lot of surprising mental and emotional health benefits. Today, we’re going to check them out.
The U.S. Food and Drug Administration has accepted the Biologics License Application (BLA) and granted priority review for emicizumab, as a once-weekly treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. Emicizumab, which is being co-developed by Chugai, Roche and Genentech, is unlike existing treatments in that it is administered subcutaneously via an injection just under the skin, instead of intravenously.
7 Ways You Can Prepare for Emergencies. Tips and resources that may help you during this hurricane season.
Alnylam Pharmaceuticals, Inc. has reported on the death of a hemophilia A patient who had been participating in a Phase 2 study for the company’s investigational therapy fitusiran.
The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “Genetics of Hemophilia.” The target audience for this presentation includes nurses and other health care professionals who are part of a U.S. hemophilia treatment center (HTC), who are interested in learning more about the genetic aspects of the condition.
Bayer applied for a Biologics License Application for BAY 94-9027, the company’s long-acting site-specific PEGylated recombinant factor VIII therapy for the treatment of hemophilia A.
Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced that the first patient received treatment in a phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of hemophilia A gene therapy programs.
The U.S. Food and Drug Administration has approved additional vial sizes for Nuwiq®, Octapharma’s recombinant FVIII therapy to treat hemophilia A patients. The new vial sizes will include 2500, 3000 and 4000 International Units (IU) and will be available in September 2017. The existing sizes include 250, 500, 1000 and 2000 IU.
What does Labor Day actually mean, and how can you use an altruistic lifestyle to get involved? Today, let's talk about the history of Labor Day and some ideas for you to join in.
Our families in the Houston area are in need of help and here is how you can assist.
Our families in the Florida area are in need of help and here is how you can assist.
Our hearts go out to our families and staff who have been impacted by Hurricane Harvey. Please know that we are here to help in any way we can.
Being kind to a stranger may seem simple, but it often requires a conscious effort to do. How can you show kindness to strangers in your life? We have 10 ideas to start.
How can you celebrate National Senior Citizens Day? We’re glad you asked. We have some ideas on how to honor senior citizens today, this week and for more times to come.
Specifically, Pfizer will expand an 11,000-square-foot plant in Sanford, North Carolina that it acquired last year when it bought Bamboo Therapeutics, a biotech company that specializes in gene therapies for certain rare diseases.
The U.S. Food and Drug Administration (FDA) has approved Mavyret (glecaprevir/pibrentasvir), a combination drug regimen for the treatment of patients with all major genotypes (1-6) of the chronic hepatitis C virus (HCV).
Complaint argues Wellmark’s withdrawal from the Iowa marketplace discriminates against protected health conditions under the ACA, including hemophilia, and its disclosure of a patient’s personal health information violates HIPAA.
Spark Therapeutics, Inc. recently released preliminary data from a phase 1/2 dose-escalation clinical trial for SPK-8011, the company’s investigational gene therapy candidate for hemophilia A.
If you want to help make a difference and support children and families in need within your community, check out our list of school supplies you can donate.
“Optimizing Transition to Extended Half-Life Factor Replacement Products,” is the latest educational opportunity to become available from Medscape. It is intended for hematologists, pediatricians, nurses, and other healthcare professionals who manage patients with hemophilia.
The activity is part of the online series, “Clinical Advances in Hemophilia: Management for Life,” whose purpose is to increase the knowledge, skills and competence of clinicians in providing individualized management of hemophilia.
The U.S. Food and Drug Administration (FDA) has approved expanded labeling for Epclusa®, a combination therapy for adults with chronic hepatitis C viral (HCV) infection.
The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “An Overview of the Background, Inheritance and Physiology of von Willebrand Disease.”
The new module is designed to enable the learner to develop an enhanced understanding of the nature of von Willebrand disease (VWD) by providing information about the discovery and evolution of knowledge about the disease. The module also includes an explanation of the production and functions of von Willebrand factor and the varied classifications of VWD.
For individuals and their families, the words “You need a new organ” are earth-shattering. Luckily, we can make a difference. Check out five reasons for you to become an organ donor.
UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA. According to a uniQure press release, the facility is designed to manufacture the company’s proprietary hemophilia B gene therapy candidate, AMT-060.
BioMarin Pharmaceutical, Inc. recently provided updated interim results from a phase 1/2 clinical study of BMN 270, the company’s investigational hemophilia A gene therapy. BMN 270 uses adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that prompt the production of the factor VIII (FVIII) protein that is deficient in people with hemophilia A. These AAVs are designed to deliver genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses.
The Committee of Ten Thousand has requested that NHF make the public aware that their board has chosen not to participate nor support any fundraising efforts for the hemophilia memorial project, led by the National AIDS Memorial Grove.
This August, we celebrate National Water Quality Month. How can you have an impact on water quality? We’ve got some ideas for the role you play in making a difference.
Vote today for the inscription that will be engraved on the Hemophilia Memorial in San Francisco, CA.
How can you live an altruistic lifestyle when negativity is on your mind? Today we've got the answers as we share six tips to help train your brain to be more positive.