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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.


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STAQ Pharma, Inc. has advised National Hemophilia Foundation and the Hemophilia Alliance and the of a typographical printing error on a few units of lot 21114255A Desmopressin Acetate 1.5 mg/mL.

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NHF STATEMENT IN RESPONSE TO 6/24 SUPREME COURT RULING




“The National Hemophilia Foundation (NHF) is aware of today’s Supreme Court decision around the reversal of Roe vs. Wade. NHF will continue to be a champion and stand firm in the fight for women’s health, health equity, and access to care for all.

The Rady Children’s Institute for Genomic Medicine (RCIGM®) recently announced the initiation of a pilot program designed to advance a diagnostic and precision medicine guidance tool known as BeginNGS™.

The U.S. diabetes epidemic and its impact on the health of tens of millions of Americans has been thoroughly reported on in recent decades, particularly as it relates to the significant impact of body mass index (BMI) as a risk. Yet little is known of its prevalence in patients with hemophilia (PWH).

Bayer has announced that they are discontinuing the recombinant factor VIII product Kogenate® FS. First approved by the U.S. Food and Drug Administration in 1993, the therapy has carried multiple indications for children and adults with hemophilia A.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of individuals with hemophilia A. Investigational breakthrough drugs are placed on a fast-track approval program and given intensive guidance from the FDA.

BioMarin recently announced that their planned timeline for the resubmission of a Biologics License Application to the U.S. Food and Drug Administration (FDA) for valoctocogene roxaparvovec, also known as Roctavian, has been pushed back to the end of September 2022 – the initial target had been June. 

June 3 is the birth date of the late physician and researcher, Dr. Charles Drew. Dr. Drew is remembered for his legacy in creating valuable techniques and processes in blood storage and blood transfusion that are still used today. He is also heralded for his efforts in health equity and racial justice in the care of African American and Black patients, and for promotion the training of Black and African American physicians.

NHF wants to know what is working well, and what isn't! How can we better serve you as an organization and community leader?


In order to become your go-to trusted resource for information on bleeding and blood disorders, NHF is asking for your feedback! Take a short survey to share your thoughts.

Click here to take the survey!

The U.S. Food and Drug Administration (FDA) has accepted CSL Behring’s Biologics License Application (BLA) for priority review of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B.

Etranacogene dezaparvovec, also known as CSL222, consists of adeno-associated virus serotype 5 (AAV5), which function as a delivery vector carrying a gene cassette with the Padua variant of factor IX (FIX). AAV5 is a variant of the type of the adeno-associated virus vectors (AAVs) investigated in multiple ongoing gene therapy clinical trials.

The next generation of researchers are continuously inspiring and fueling NHF’s renewed focus on research. Dr. Olubusola (Bosula) Oluwole is one of the first-ever recipients of the new Jeanne Marie Lusher (JML) Diversity Fellowship.

NHF’s newest research fellowship program is named in honor of Dr. Jeanne Marie Lusher, a distinguished clinician and researcher whose career focused on inhibitor development in factor VIII patients.

Meet NHF's Education Specialist, Sonia Roger! As one of NHF's most tenured employees, Sonia is a vital part of the NHF community and loves giving back to families and patients. Learn more about her role at NHF!

Tell us a little bit about yourself and your role at NHF.

The issue of poor bone health represents a significant public health concern in the U.S. population, including for individuals with hemophilia who are at increased risk for low bone mineral density (BMD) and bone fractures.

Newly published data from a phase 3 clinical study suggests that the recombinant factor VIIa bypassing agent (BPA) eptacog beta was used to safely and effectively treat and control bleeding in children with hemophilia A or B with inhibitors, 12 years of age or younger.

NHF’s Medical and Scientific Advisory Council (MASAC) recently issued six new documents, all of which were adopted by NHF’s Board of Directors on April 27, 2022.

 

A group of investigators are embarking on a new research program designed to unpack some of the outstanding fundamental questions associated with current approaches to gene therapy for hemophilia A.

NHF Announces Spring Webinar Lineup

FOR IMMEDIATE RELEASE

Media Contact:

Ilana Ostrin
iostrin@hemophilia.org

212-328-3769

April 17, 2022

In advance of World Hemophilia Day this year (April 17), NHF sat down with historian Dr. Helen Rappaport to learn more about how hemophilia impacted the Romanov family, and its lasting impact on world history. Read the interview transcript, or watch the video recording.

The UK-based biotechnology company Freeline recently announced that the first U.S. patient has been dosed in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for the treatment of hemophilia B. FLT180a is developed with the company’s proprietary adeno-associated viral (AAV) vector and its protein shell capsid, known as AAVS3, to deliver the genetic material that generates therapeutic factor IX levels in hemophilia B patients.

NHF sat down with NYLI member Patti W. to hear more about why she joined NYLI and what others should consider when filling out their application. Visit www.hemophilia.org/nyli">NYLI to learn more and fill out your own application.

NHF sat down with NYLI member Patti W.

Ahead of National Doctors Day (March 30), NHF CEO and hematologist

We are excited to announce that NHF’s Resource Center HANDI has launched a new microsite to coincide with Bleeding Disorders Awareness Month! The site includes many frequently asked questions (FAQs) received by HANDI.

Student Hajar Abuisef sat down with NHF to discuss her involvement in the Twitter account, Dr. Period Hackers. Read these Q&As, and watch the video to learn more!

How did you come to be a part of Dr. Period Hackers?

Dr. Bethany Samuelson Bannow sat down with NHF to discuss her co-authored Twitter account, Dr. Period Hackers. Read these Q&As and watch the video to learn more!

What inspired you to start Dr. Period Hackers?

Dr. Marybec Griffin graciously sat down with NHF for an informative dialogue to share information on her co-authored Twitter account, Dr. Period Hackers.

It’s almost that time of year!

Awareness month campaign encourages community to raise their voices and share what is most important and relevant to their conditions throughout March

Dr. Johnny Mahlangu gracious sat down with NHF's CEO Dr. Len Valentino for an informative dialogue to honor Black History Month and World Hemophilia Day. Read these Q&As and watch the video to learn more!

Black History Month and World Hemophilia Day Discussion with Dr. Johnny Mahlangu

As the eyes of the world fix upon Ukraine, I am shocked and saddened to see heartbreaking images emerge showing injured civilians staggering through the streets and towns in the east of the country following Russian bombing over the last several days. This is an unprecedented atrocity for which there is no justification. Russia’s decision to attack will bring catastrophic consequences to Ukraine. We stand in solidarity with the people of Ukraine and with every freedom-loving democracy in condemning Russia’s military actions.

In 2020 NHF began an exciting collaboration with a number of blood disorder groups who receive funding from the Centers of Disease Control and Prevention.

Please click this link to read an update from BioMarin on a phase 3 trial.

Takeda recently announced that the U.S. Food and Drug Administration (FDA) has approved VONVENDI® for routine prophylaxis to reduce the frequency of bleeding episodes in adults with severe Type 3 von Willebrand disease (VWD) receiving on-demand therapy. Type 3 VWD is the rarest and most severe form of the disease.

VONVENDI® is the only currently available recombinant von Willebrand factor therapy on the market.

Medscape has launched a new enduring activity designed to enhance clinicians’ awareness of gene therapy for hemophilia and increase their confidence when it comes to the eventual integration of these paradigm-shifting therapeutics into the clinical care of patients.



The program, which was developed through a partnership between Medscape and NHF, is intended for an international audience of hematologists/oncologists, pediatricians, hematology/oncology nurses, nurse practitioners, and physician assistants.

This piece is a joint effort by Amy Niles, executive vice president at the PAN Foundation, and Kollet Koulianos, vice president of payer relations at the National Hemophilia Foundation.



 


Each year on January 1, health plans reset for millions of Americans with serious and complex illnesses, restarting the slow and often expensive process of working toward their annual deductible and maximum out-of-pocket limit. But for people with co-pay accumulator policies built into their health insurance plans, there’s a much steeper hill to climb.

BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy.

WASHINGTON, D.C.— December 31, 2021 — In response to additional rulemaking for the Centers for Medicare & Medicaid Services’ (CMS)

FOR IMMEDIATE RELEASE

Media & Community Inquiries

Brett Spitale, VP of Advancement

Dec. 31, 2021 – New York, NY – NHF is deeply saddened to announce that former NHF CEO and community member, Val Bias, passed away suddenly on Thursday, Dec. 30.

Val and I first got to know one another 30 years ago, when we were opposing one another in an election for president of the National Hemophilia Foundation (US). I was fighting for HIV/AIDS advocacy from within, from the Board, and Val was part of a more activist group that was impatient the NHF wasn’t doing enough, fast enough. He and I came together, realized our goals were the same, and also recognized we were so much stronger together.

Results of a recently published study suggest that intense physical activities, particularly those that include a high collision-risk, can increase bleeding risk in individuals with severe or moderately severe hemophilia A. Investigators also found that increasing the time between the administration of factor (FVIII) and physical activity also heightened bleeding risk.


Sigilon Therapeutics recently announced a “strategic reprioritization,” as the company shifts focus to their non-hemophilia, investigational therapies, including those for MPS-1 and diabetes. This closely follows recent setbacks associated with their phase 1/2 clinical study of SIG-001 in individuals with severe or moderately severe hemophilia.

Interested in working in the hematology field, but don’t think that medical school is right for you? Consider an allied health field, like genetic counseling or physical therapy. Recently, NHF sat down with Mitch Starkman, a physical therapist, and Stefanie Dugan, a genetic counselor, to discuss their career paths and professional experiences. Read these Q&As and watch their respective videos to learn more! 

 

The onset of childbirth and the postpartum period are times when women with von Willebrand disease (VWD) are at an increased risk for excessive bleeding, exposing them to further, and in some instances, serious complications. While there exist therapies with VWD-specific indications, it is not uncommon for these patients to still experience excessive bleeding while receiving treatment. These scenarios are challenging as there is sparce clinical data and a subsequent lack of clear guidance on the optimal management of bleeding in these particular settings.

uniQure and CSL Behring recently announced that etranacogene dezaparvovec, an investigational hemophilia B gene therapy currently in clinical trials, has achieved a primary endpoint of “non-inferiority” in annualized bleeding rate (ABR) 18-months post administration. The non-inferiority component of a clinical trial measures whether the investigational therapy is not worse/less efficacious than a currently available product. In this case, etranacogene dezaparvovec is being compared to baseline factor IX prophylactic replacement therapy as part of the phase III HOPE-B pivotal trial.

PRESS RELEASE





FOR IMMEDIATE RELEASE

MEDIA CONTACT:

Sigilon Therapeutics today reported a new development in the company’s phase 1/2 clinical study of SIG-001 in individuals with severe or moderately severe hemophilia. This follows the events of this past summer when the U.S. Food and Drug Administration (FDA) placed a clinical hold on the trial due to an unwanted inhibitor response in one of the three patients participating in the study.

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