News

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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?


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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

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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

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