4696 W. Overland Road, Suite 234
Boise, Idaho 83705

Newsroom

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


Shared decision making (SDM), the concept by which patients and healthcare providers work closely together to arrive at personalized treatment-related decisions, can have significant positive impacts for individuals with hemophilia.

A new paper published in The Journal of Haemophilia Practice advances the concept of shared decision making (SDM) between the hemophilia patient and their healthcare provider (HCP).

Inspiration is a fickle thing. It comes and goes with little warning. The good news is, you can always reignite inspiration when you’re feeling uninspired.

A new article, “Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia,” was published in the Journal of Clinical Medicine (JCM).



In this review, the authors discuss some of the primary considerations relevant to investigational gene therapies that employ adeno-associated viral (AAV) vectors, with particular focus on immunogenicity and genotoxicity – the former denotes the ability of a foreign substance to trigger an immune response, while the latter refers to a substances ability to damage genetic material.

FOR IMMEDIATE RELEASE

Media Contact

Ilana Ostrin

iostrin@hemophilia.org

212-328-3769

While summer is a time of fun, it can also be a time to help mold amazing qualities, like giving back to the community. Ask your children to choose an activity that also gives back

If you’re able, use World Blood Donor Day as a time to give back and save lives by donating blood. If you aren’t able to safely do so, consider helping out in one of the various other ways to help spread awareness.

FOR PUBLIC COMMENT - JUNE 3, 2021

Weather across the country is heating up, and it’s the perfect time to get out and enjoy mother nature. As it turns out, when it comes to spending time outside, the benefits go far beyond the fresh air.

BioMarin recently reported key findings from preclinical studies of their investigational, gene therapy Roctavian (valoctocogene roxaparvovec), which was developed for the treatment of adults with severe hemophilia A. The findings were presented at the American Society of Gene & Cell Therapy (ASGCT) Virtual Meeting, which was held May 11-14, 2021.

While the future is uncertain, mankind remains resilient. We’ve learned to adapt to a “new normal” and are slowly making our way back to activities that we set aside for more than a year. A lot has changed, and some of the new techniques and processes surrounding fundraisers, volunteering and fundraising are here to stay.

The National Hemophilia Foundation (NHF) today, in conjunction with the American Kidney Fund (AKF), Arthritis Foundation, and American Autoimmune Related Diseases Association (AARDA), released findings from a new national online survey of patients and caregivers that illustrates the alarming challenges t

Important research using data from the My Life, Our Future (MLOF) Research Repository Phase One has been published in Frontiers in Medicine.

The upcoming Memorial Day holiday is all about honoring those who paid the ultimate sacrifice for our country. This is the perfect holiday to exercise taking a day “on” instead of a day off.

“Scholarship season” is now in full swing, time for an appointment reminder!

To lead a fulfilled life, being healthy is about much more than just the physical. It involves your mental well-being, too. 

uniQure recently announced that their commercialization and license agreement with CSL Behring  for their investigational hemophilia B gene therapy was finally closed on May 5, 2021 – the agreement had been pending the completion of a series of antitrust reviews in the U.S., Australia, and the U.K.

Findings from a recently published study in the Journal of Anesthesia suggest that pregnant patients with hemophilia whose factor VIII or IX levels drop below 50% at the time of receiving neuraxial anesthesia are more likely to experience postpartum complications.

You don’t have to choose just one cause or organization to support. You can provide support for various causes and various organizations. Take the time to figure out what feels right for you and your own goals for doing good in the world.

Hemophilia Treatment Centers (HTCs) across the United States are participating in the 2021 National HTC Patient Satisfaction Survey. If you, or someone you care for, had contact with the HTC in 2020, they want to hear from you! You can take the survey online at www.htcsurvey.com, or fill out the paper survey you received in the mail. The survey closes on June 30, 2021.

NHF's Wednesday Webinars for May 2021 will focus on Women's Health Month,  featuring topics  of interest to both patients and healthcare providers who are interested in issues faced by women with inheritable blood disorders.

May Webinars

All webinars are from 2:00pm-3:00pm ET.

May 5, 2021

VWD Guidelines: Diagnoses

Speaker: Robert Sidonio, MD

 

May 12, 2021

uniQure recently announced that the U.S. Food and Drug Administration (FDA) has removed a clinical hold on their hemophilia B gene therapy program. The FDA had determined that uniQure satisfactorily addressed all issues raised by the agency that were related to a single patient diagnosed with hepatocellular carcinoma (HCC), a common form of liver cancer.

May is recognized as National Recommitment Month. It’s a time of renewal and even to build new habits or relationships. What will you recommit to this month?

Kevin Mills, PhD has joined the National Hemophilia Foundation as chief scientific officer.

A graduate of University of Colorado Boulder, MIT, and Harvard Medical School, Dr. Mills will be responsible for overseeing NHF’s research strategy, including developing and expanding the foundation’s research capabilities, and setting research goals.

Dear NHF Family,

Like everyone else, I was closely following the trial and eventual verdict in the case of Derek Chauvin for the murder of George Floyd, and I know that many of you were anxiously awaiting as well. Mr. Floyd’s death was senseless and an ugly act of violence.

It was my hope that the jury would evaluate the facts and render a a fair verdict, and in turn, pave the way for greater accountability at all levels of our society, including law enforcement.

Now that the world is showing signs of getting back to a sense of “normalcy,” use this Earth Day as a reminder that when we all work together, we can make a difference.

You have the power to continue to do good in the world and be the positive change, even with everything going on around us.

If you’re looking for a reason to get out there and do more good, look no further. April has been named Global Volunteer Month as a time to bring people together to volunteer from across the world.

NHF's Wednesday Webinars for April 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

April Webinars
All webinars are from 2:00pm-3:00pm ET.

April 7th, 2021
What is the Burden of Problem Joints with Hemophilia?

Speaker: Paul McLaughlin

April 14th, 2021
WFH Humanitarian Aid Program

Speaker: Assad Haffar

The American Rescue Plan (ARP), signed into law on March 11, 2021, provides new opportunities to get health insurance coverage, and/or to reduce the cost of your premiums. Review your coverage now to make sure you are maximizing your benefits under the new law.

 

uniQure recently announced the results of a comprehensive investigation into a case of hepatocellular carcinoma (HCC) diagnosed in one patient participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, the company’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.

uniQure recently announced the results of a comprehensive investigation into a case of hepatocellular carcinoma (HCC) diagnosed in one patient participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, the company’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.

While times are undoubtedly hard, there is hope on the horizon. Use April’s National Month of Hope as a reminder to be a light in somebody’s life this month, including your own!

When you feel good, you’re more likely to do good in the world and at work. Make sure you’re able to set boundaries to help improve your work-from-home life.

To the members of the inheritable blood disorders community,

Let me begin with a word for the thousands of members of our community – patients, providers, partners, family, and friends – who are also members of the Asian and Pacific Islander community:

We would not be the National Hemophilia Foundation without you.

You may not win the lottery tomorrow, or ever for that matter. But what we’re talking about is a different kind of ‘gold’ to make you feel rich in other ways. Check out these ways to feel fulfilled in your everyday life and to make each day golden.

The National Hemophilia Foundation (NHF) announced that Keri L. Norris, PhD, MPH, MCHES, has joined NHF as  vice president of health equity, diversity, and inclusion. Dr. Norris has more than 20 years of experience in health equity, public health, and social justice, with a particular emphasis on health disparities. Her role will be to create a health equity framework to integrate into NHF programs and services and develop culturally and linguistically appropriate programs and services to address disparities in outcomes within our community.

The National Hemophilia Foundation (NHF) announce plans for a NHF State of the Science Research Summit to bring together the inherited bleeding disorders community for a virtual conference to identify and accelerate research progress in the areas of greatest need for affected individuals and their families. The event is scheduled to be held virtually September 12-15, 2021.

In Genentech’s ongoing efforts to transparently communicate with the hemophilia community, we are sharing two updates to the Hemlibra label that were requested by the FDA. These changes, which were accepted and implemented on March 10, 2021, impact the Warnings and Precautions (sections 5.1 and 5.2) and Clinical Pharmacology (section 12.3) sections of the label.​ Importantly, the overall benefit/risk profile of Hemlibra remains positive.

The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) issued three new documents, all of which were adopted by NHF’s Board of Directors on March 4, 2021.

The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) issued three new documents, all of which were adopted by NHF’s Board of Directors on March 4, 2021.

There are those who enjoy crunching numbers, and those who would love to be doing anything else. With March underway and April just around the corner, you may be dreading a certain time of year that’s all but looming—tax time.

The National Hemophilia Foundation (NHF) is refocusing its mission statement to reflect a renewed emphasis on research and an expanded scope to include inheritable blood disorders.

The newly adopted mission statement reads:

The mission of the National Hemophilia Foundation is dedicated to finding cures for inheritable blood disorders and to addressing and preventing the complications of these disorders through research, education, and advocacy, enabling people and families to thrive.

The National Hemophilia Foundation is now accepting nominations for its Awards of Excellence. These awards honor members of the bleeding disorders community who have made outstanding contributions to improving the lives of people with bleeding disorders through treatment and care, advocacy, and raising awareness.

The Children’s National Hospital and Takeda recently announced the creation of a new, first-of-its-kind program to help standardize and streamline the process of diagnosis and care for individuals with rare diseases.

It’s impossible to put the best version of yourself out into the universe if you’re already starting at a disadvantage. Make sleep a priority and see the health benefits that affect your overall wellbeing.

NHF's Wednesday Webinars for March 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

March Webinars
All webinars are from 2:00pm-3:00pm ET.

 

March 3, 2021

Cellular Therapies for Hemophilia 

Speakers: Amy Shapiro, MD, Indiana Hemophilia & Thrombosis Center, Inc. and Deya Corzo, MD, Sigilon Therapeutics

 

The U.S. Food and Drug Administration (FDA) has partially rescinded a pair of supplemental Biologics License Applications (BLAs) the agency had granted, in error, for two recombinant factor IX products, BeneFIX® (Pfizer) and IXINITY (Aptevo). The supplemental BLAs in question were both approved by the FDA in 2020 and represented additional indications that ultimately ran against exclusivity rights of another product.   

Background:

4696 W. Overland Road, Suite 234
Boise, Idaho 83705

© Idaho Chapter of the National Hemophilia Foundation 2021

Crafted by Firespring