Newsroom


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

Be sure to check back regularly to get our latest news updates.


Pfizer to Build Gene Therapy Facility in North Carolina

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.

FDA Approves New Combination Therapy for all Major Hepatitis C Genotypes

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).

Leading Bleeding Disorders Advocacy Organizations File Hemophilia Discrimination Complaint Against Wellmark Blue Cross and Blue Shield

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 Reports Preliminary Data from Hemophilia A Gene Therapy Trial

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.

“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.

FDA Expands Labeling for Epclusa® to Include Co-Infected Patients with HCV/HIV and Warning for HBV Reactivation

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.

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.

Choose the Inscription on the Hemophilia Memorial

Vote today for the inscription that will be engraved on the Hemophilia Memorial in San Francisco, CA.

The program will allow bleeding disorders camp staff learn from one another and share best practices.

The Partners in Bleeding Disorders Education Program recently launched an updated version of “Dear Diary: The Importance of Infusion Logs in Home Infusion Therapy.” This activity aims to enable the learner to apply knowledge of the benefits and applications of paper and electronic infusion logs to support their accurate use among hemophilia patients engaged in home infusion.

Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme announced positive results from an ongoing Phase 2 open label extension (OLE) study with fitusiran in patients with hemophilia A and B, both with or without inhibitors. Details from the study were presented at 26th International Society on Thrombosis and Haemostasis Meeting, July 8-13, 2017, in Berlin, Germany.

Shire Submits IND Application for Hemophilia A Gene Therapy Candidate

If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.

Shire has announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A.

Genentech Announces Progress in Two Phase III Emicizumab Studies

Results showed a statistically significant and clinically meaningful reduction in bleed rate, reports states.

Bleeding Disorder Organizations Unite in Opposition to BCRA

NHF, HFA, the Hemophilia Alliance and Coalition for Hemophilia B are united in their opposition to BCRA because of its damaging impact on our community and urge the Senate to reject these harmful policies.

The Senate Bill (BCRA) Will Hurt the Bleeding Disorders Community

Call TODAY and tell your Senator to vote NO on BCRA!

 

The Partners in Bleeding Disorders Education Program recently relaunched “Understanding Hemophilia,” the purpose of which is to enable the learner to understand the three major steps in normal hemostasis and the basics of treatment and management of patients with hemophilia.

The target audience includes nurses and comprehensive care staff in the U.S. hemophilia treatment center (HTC) network who are responsible for the clinical management and/or education of individuals with hemophilia.

The faculty for this module includes:

Green Park Collaborative, National Hemophilia Foundation, McMaster University, Announce Launch of CoreHEM Project

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for REBINYN®, the company’s recombinant, GlycoPEGylated therapy for the treatment of adults and children with hemophilia B (factor IX deficiency). A BLA is a request to the FDA for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.

One of Shire’s investigational therapies, known as SHP656, failed to show significant efficacy in a phase I/II clinical study. The therapy was being developed as an extended half-life treatment for hemophilia A using proprietary technology from Xenetic Biosciences.

A Million Dollar Patient in Perspective

A wider perspective on the "burden" hemophilia patients place on health plans.

The Nationwide Children’s Hospital Pediatric Hematology Division along with its affiliated institution, The Ohio State University College of Medicine, are jointly hosting an upcoming educational training on inhibitors. The Center for Advanced Training in Hemophilia and Inhibitor Management (CATHIM) workshop will focus on the clinical care of patients with hemophilia and inhibitors.

The Partners in Bleeding Disorders Education Program recently relaunched “A School Nurses Guide to Bleeding Disorders: von Willebrand Disease.” The purpose of this activity is to enable the learner to obtain knowledge about von Willebrand disease (VWD), its treatment, and any accommodations that must be made to ensure safe and positive learning environment to the student with VWD.

NHF's update on the CBO scoring of the AHCA; President Trump's proposed budget, and how it will all affect you.

9-Year-Old Girl Raises $40,000 at NHF's Red Tie Soiree

NHF hosted a successful and festive benefit gala, the Red Tie Soiree, on Thursday, May 18, 2017, at Current at Chelsea Piers - Pier 59 in New York City.

Medscape Education recently launched a new activity, the focus of which is a report from the journal Emerging Infectious Diseases (EID) on surveillance as it relates to two unique cases of patients deceased and diagnosed with sporadic type Creutzfeldt-Jakob Disease (sCJD) in the United Kingdom (UK). sCJD is the most common type in a group of rare neurodegenerative diseases characterized by abnormal prion proteins that form sponge like holes in the brain tissue, resulting in death. sCJD arises in patients with no known risk factors for the disease.

The Partners in Bleeding Disorders Education Program recently relaunched an educational activity focused on clinical research at hemophilia treatment centers (HTCs) entitled “The Basics of Research at Hemophilia Treatment Centers.” The purpose of the module is to enable learners who care for patients with bleeding disorders to participate in research, interpret, and apply specialty protocols and outcome findings effectively.

Raise Your Voice and Support Patient Protections!

NOW is the time to call your representative to tell him or her to vote NO on AHCA as revised by the MacArthur amendment.

The BloodCenter of Wisconsin (BCW) recently announced the launch of a new and more sensitive test for von Willebrand disease (VWD), a genetic disorder characterized by either a qualitative or quantitative flaw in von Willebrand factor (VWF). Milwaukee-based BCW is a not-for-profit organization that specializes in blood services, organ, tissue and marrow donation, diagnostic testing, medical services and research.

The Partners in Bleeding Disorders Education Program is offering a new educational module designed to familiarize staff of the 140+ federally recognized U.S.

Bilingual Volunteers Needed

You will help guide people to services and support offered by bleeding disorders organizations and HTCs.

NHF Focus on Physical Fitness

Check out the brand new resources from NHF to help you get active!

Genentech, a member of the Roche Group has announced interim results from their phase III HAVEN 2 study, designed to evaluate the prophylaxis use of emicizumab (ACE910) in children less than 12 years of age with hemophilia A and inhibitors to factor VIII (FVIII).

Grifols Increases Commitment of Factor VIII Donations

The company will donate a minimum of 140 million international units (IU) of factor VIII to the WFH Humanitarian Aid Program over the next five years.

In Honor of World Hemophilia Day, Enroll in MLOF!

MLOF is nearly one-third of the way towards its goal of enrolling 2,000 confirmed carriers in the MLOF Research Repository by the end of 2017.

Hemophilia B Trial Gene Therapy Results Promising

Spark reports that all participants have experienced “consistent and sustained increases in factor IX activity” following administration of SPK-9001.

Advocate for the Community in Your Hometown!

Your Senators and Representatives are coming home for the next two weeks. You can advocate for the community in your own hometown with this guide.

The University of California at San Diego (UCSD) School of Medicine is currently offering a CME-accredited course called “Musculoskeletal Ultrasound in Hemophilia.” The purpose of the two-and-a-half-day program is to provide hands-on training in musculoskeletal ultrasound (MSKUS) as it relates to hemophilic joint disease. The program is being offered multiple times in 2017 (next trainings are scheduled for June 28-30, August 16-18 and October 18-20) at the Hemophilia & Thrombosis Treatment Center at UC San Diego in California.

The Partners in Bleeding Disorders Education Program is offering a new educational module to address the unique challenges facing individuals and families affected by inhibitors. The target audience for this activity includes psychosocial professionals, nurses and other health care providers at hemophilia treatment centers who are involved in the care of persons with congenital hemophilia who have an inhibitor to clotting factors VIII or IX.

The module is designed to meet the following educational objectives:

Scientists from the Center for Cardiovascular Research at the University of Hawaii (UH) have developed a novel approach to hemophilia gene therapy that employs microbubbles and an ultrasonic beam to deliver the treatment.

NHF CEO Val D. Bias shares this thoughts on the success of Bleeding Disorders Awareness Month 2017.

HTC Program Funding Needs Your Help!

We asked for your help last week and your rocket it! Your help is needed again -- HTC funding could be in jeopardy!

 

Let the Stars in Our Community Shine!

Help the heroes in our community get the recognition they deserve!

Guías Culturales are culturally intelligent volunteers who are the path to service to the bleeding disorders community, acting as “guides” to diverse members of the bleeding disorders community.

Josh Gordy is a National Football League free-agent.  Josh created these scholarships in honor of his nephew, Nolan, who has severe hemophilia A. Applicants must be individuals diagnosed with hemophilia who are entering or attending an accredited 2- or 4-year undergraduate institution in the United States for the 2017-18 school year.

Award amount: $1,000

Number of awards: 2

Registration opens: 4/24/2017

Registration closes: 5/31/2017