Newsroom


If you want to be in the know about what’s going on at our organization, you’ve come to the right place!


The rate of attendance to scheduled clinic visits have been linked to health outcomes in several chronic disease groups such as diabetes, hypertension, HIV and congenital heart disease. Higher clinic attendance rates are generally associated with better outcomes in these patients. However, the potential effects of non-attendance (NA) for individuals with bleeding disorders is not well understood.

Pfizer Initiates First Stage of Phase 3 Hemophilia B Gene Therapy Trial

The first stage will collect data that will be eventually used to evaluate the efficacy and safety of the investigational hemophilia B therapy.

There’s more to Global Hug Your Kids Day than just giving your kiddos a hug or two. If you want to celebrate today, here are some fun ideas to get you started.

Setting goals is simple. We’ve all heard different phrases, acronyms and ways to properly set a goal for life. However, accomplishing goals can be a whole different task.

Advanced Considerations for the Recognition, Appropriate Diagnosis, and Timely Management of von Willebrand Disease and Other Bleeding Disorders among Women in Non-Hematology Health Care Settings

Take part in a survey to shape VWD Guidelines. We want to hear from people with VWD, people who care for those with VWD, or clinicians and other healthcare professionals who treat people with VWD.

Do you want to make a difference and give back to your community this holiday week? Check out our list of volunteering ideas for you to try on the Fourth of July.

A recent article appearing in the journal Haemophilia, includes updates on the My Life Our Future (MLOF) project, which was established in 2012 to offer U.S. hemophilia patients ready access to free genotyping, a type of genetic testing that is often hard to access, expensive and not covered by insurance. MLOF is a partnership between the American Thrombosis and Hemostasis Network (ATHN), the National Hemophilia Foundation (NHF), Bloodworks Northwest and Bioverativ (formerly Biogen).

Medscape has launched the latest educational activity in their “Clinical Advances in Gene Therapy for Hemophilia” series, which is presented through a collaboration between Medscape, the National Hemophilia Foundation, the European Haemophilia Consortium and the World Federation of Hemophilia. The program is supported by an independent educational grant from BioMarin.

Fortunately, there are a few ways and practices to grow in your communication skills. Here are healthy habits you can try to become a strong communicator.

The goal of this effort is to create and maintain state-of-the-art guidelines on the diagnosis and management of VWD.

Catalyst Biosciences, Inc. recently released an update relevant to an ongoing Phase 1/2 clinical trial for CB 2679d/ISU304, the company’s investigational therapy for the treatment of patients with severe hemophilia B/factor IX (FIX) deficiency. Catalyst develops its therapies using engineered proteases, enzymes that increase the rate of chemical reactions within cells. These proteases can target the proteins associated with certain underlying diseases and conditions, such as FIX.

Because of their lower factor levels, hemophilia patients are generally thought to be better protected from cardiovascular disease (CVD). To determine whether data would support this long-held assumption, a team of investigators enrolled patients from 19 U.S. hemophilia treatment centers (HTCs) in the “CVD in Hemophilia” study. The lead author of the study was Barbara Konkle, MD, Professor of Medicine in the Division of Hematology at the University of Washington School of Medicine in Seattle, WA.

HTC Studies Cardiovascular Disease in Hemophilia Patients

Investigators looked into whether having hemophilia protected patients against cardiovascular disease.

Update on Latest ACA Lawsuit

More information on how the current ACA lawsuit could affect the bleeding disorders community.

If you’re considering volunteering with a cause you care about in the community, consider the benefits you can receive. Here are the top eight benefits of volunteering.

Grifols Announces Recall of One Lot of Profilnine. This recall is being conducted as a precautionary measure.

Caring for animals is an important part of living an altruistic lifestyle. Today we explore how you can live more altruistically with the animals in your day-to-day life.

Genentech Announces the Launch of New Safety Website

The new web portal for patients and caregivers is intended to provide timely and accurate information on targeted serious adverse events of interest for HEMLIBRA.

This priority review is specifically for adults and children with hemophilia A without FVIII inhibitors.

Roche Holding AG announced that the U.S. Food and Drug Administration (FDA) has granted “priority review” to HEMLIBRA® a subcutaneously administered therapy currently being investigated for the treatment of patients with hemophilia A, without factor VIII (FVIII) inhibitors. The therapy, which is co-developed by Genentech, Chugai and Roche, is already FDA-approved for routine once-weekly prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors.

CSL Behring recently announced that the US Food and Drug Administration (FDA) has approved a new 3500 IU (international unit) vial size for the company’s recombinant factor IX (rFIX) product Idelvion®. Idelvion combines or “fuses” recombinant factor IX (rFIX) with albumin, a protein found in blood plasma that helps factor IX stay active in the bloodstream longer.

uniQure presented new hemophilia B clinical trial data on May 19, 2018 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Chicago, IL. The data showed its hemophilia B investigational gene therapy candidate AMT-060 could be successfully delivered to patients with certain pre-existing antibodies. Individuals testing positive for “anti AAV5 neutralizing antibodies” (NABs), are often excluded from trials, as they were thought to impede the delivery of some gene therapies.

If you try to live altruistically, then combating your own loneliness can play a vital role in how you serve others. Here’s how to overcome loneliness in everyday life.

Our dedicated providers and inspiring volunteers and advocates can be recognized with an Award of Excellence!

Our dedicated providers and inspiring volunteers and advocates can be recognized with an Award of Excellence!

New Study on Aging, VWF Levels and Bleeding Risk

Investigators looked to establish whether VWF levels increase as patients with VWD age, and if so, whether this can be associated with lower risk for bleeding.

NHF's Dawn Rotellini Elected to WFH Board of Directors

"It has become my passion to make a difference for people throughout the world. We all need access to treatment and care and education.”

How can you continue to honor and respect the service men and women dedicated to protecting this nation? Here are six ways to show your support this Memorial Day.

Spark Sees Continued Progress in Hemophilia B Gene Therapy

The overall annualized bleeding rate for all 15 participants was reduced by 98% to an annual rate of 0.2 bleeds per patient.

In the Phase III HAVEN 3 study, patients who received HEMLIBRA prophylaxis every week or every two weeks experienced a 96% and 97% reduction in treated bleeds.

Novo Nordisk Announces BLA Submission for Extended Half-Life FVIII Therapy

Genentech  informed NHF of a postmarketing fatality that has occurred in an elderly patient who received treatment with emicizumab- kxwh (Hemlibra) for acquired hemophilia A.

Mentoring a child helps them to see unlimited possibilities for the future, learn something new and most importantly, realize that somebody cares about them.

According to a recent study, most post pubertal girls and adolescents who experience heavy menstrual bleeding are not screened for von Willebrand disease (VWD), despite years of recommendations from the American College of Obstetricians and Gynecologists. Investigators came to this conclusion after conducting a retrospective analysis of a national claims database of public and privately insured individuals from 2011-2013.  

Study Shows VWD Testing Remains Low

Most postpubertal girls and adolescents who experience heavy menstrual bleeding are not screened for von Willebrand disease (VWD), despite years of recommendations from the American College of Obstetricians and Gynecologists.

Prophylaxis study results for boys with severe hemophilia A

Researchers investigated an approach to prophylactic therapy in which patients began by receiving weekly infusions of standard half-life recombinant factor VIII and ultimately transition to “tailored frequency-escalated prophylaxis."

Honoring the Past, Embracing the Future

NHF's Red Tie Soiree paid tribute to longtime advocates for the bleeding disorders community while looking forward to the future of research and treatment.

While it might seem simple to say talents can help others, actually identifying your talents and putting them to good use can be a little bit more difficult.

Authors of an article published recently in the Journal of Thrombosis and Haemostasis (JTH) looked at the treatment of individuals with factor X deficiency (FX) with a human plasma-derived concentrate (pdFX). FX is one of the rarer bleeding disorders with an estimated incidence of 1 in 500,000 to 1 in a million and though they represent a very small patient population the symptoms associated with FX are no less problematic, especially if not properly treated.

Advanced Considerations for the Recognition, Appropriate Diagnosis, and Timely Management of von Willebrand Disease and Other Bleeding Disorders among Women in Non-Hematology Health Care Settings

The study looked at the treatment of individuals with factor X deficiency with a human plasma-derived concentrate.

Joan Gill, MD

Dedicated clinician and distinguished researcher passed away on May 9, 2018.

NHF 70th: Unite for Bleeding Disorders

Our Unite for Bleeding Disorders walks bring the community together in a way that honors the past while looking forward to the future.

Medicaid

CMS rejects time limit proposal in Kansas; allows work requirements in several other states.

Healthcare professionals who manage patients with hemophilia can enhance their knowledge of gene therapy for hemophilia.

Medscape has launched a new education program for providers seeking to enhance their knowledge of gene therapy for hemophilia. “Clinical Advances in Gene Therapy for Hemophilia,” is presented through a collaboration between Medscape, the National Hemophilia Foundation, the European Haemophilia Consortium and the World Federation of Hemophilia. The program is supported by an independent educational grant from BioMarin.

coreHEM Publishes Core Outcome Set for Hemophilia Clinical Trials;

coreHEM has produced the first set of guidelines recommending a specific, minimum set of outcomes to include in hemophilia gene therapy clinical trials.

Mary Lesh, RN, MS, CPNP

In recognition of National Nurses Week, Mary Lesh, RN, MS, CPNP speaks of the care HTC nurses provide to people with bleeding disorders.

If you strive to live an altruistic lifestyle, there are plenty of gifts you can give your loved one on Mother’s Day that also give back to others in the community.

NHLBI Factor VIII Inhibitor Workshop Registration Now Open

The workshop aims to solicit hemophilia community-wide input into a coordinated national blueprint for future research on factor VIII immunogenicity and factor VIII inhibitor prevention and eradication.

A study conducted at the Los Angeles-based Orthopaedic Institute for Children (OIC) suggests that enhanced education on intravenous self-infusion of factor therapy can significantly reduce prosthetic joint infections for hemophilia patients. The lack of adherence to proper self-infusion protocol, including the practice of universal precautions, can put patients at a higher risk for infections.   

Knee joint

OIC study suggests that enhanced education on intravenous self-infusion of factor therapy can significantly reduce prosthetic joint infections for hemophilia patients.