News


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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.

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.

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 center (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.

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

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.

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.

Investigators from the Hemophilia Center of Western Pennsylvania (HCWP) recently looked at the effect of aging on von Willebrand factor (VWF) levels in patients with type 1 von Willebrand disease (VWD). The authors of the article were Craig D. Seaman, MD, MS and Margaret V. Ragni, MD, MPH. Seaman is Assistant Professor of Medicine, Division of Hematology/Oncology at HCWP. Ragni is Professor of Medicine and Clinical Translational Science, Department of Medicine, Division of Hematology/Oncology University of Pittsburgh Medical Center. She is also the Director of HCWP.

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.”

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.

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.

A group of Canadian researchers recently published findings from a clinical study on the use of prophylaxis with a standard half-life recombinant factor VIII (SHL-rFVIII) therapy to treat boys with severe hemophilia A. They investigated an approach to prophylactic therapy in which patients began by receiving weekly infusions of SHL-rFVIII and ultimately transition to “tailored frequency-escalated prophylaxis.” The lead investigator of the study was Professor Brian M Feldman, MD of the Division of Rheumatology at The Hospital for Sick Children in Toronto.