Hawaii Chapter - NHF
Phone 808-284-7417

NEWS


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 and Sangamo have announced that recruitment has re-opened for the phase 3 AFFINE study for the investigational gene therapy giroctocogene fitelparvovec. This clinical study is evaluating the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) participants with moderately severe to severe hemophilia A.

FOR IMMEDIATE RELEASE                                                                                

Ilana Ostrin    

Senior Director of Public Relations and Communications   

iostrin@hemophilia.org  

212-328-3769  

 

Upcoming topics include emerging research, key political issues, and more.

 

Patti joined NHF’s team after becoming a member of NYLI in 2018. Learn more about her experience!

Joseph expanded his role at his local NHF chapter with the skills he learned through NYLI. Learn more about his journey!

Dejah uses her NYLI experience to lead others as she pursues her master’s degree in biology. Learn about her story here!

This week, a Peoria Illinois jury awarded reputation-related damages in favor of Dr. Osvaldo Wesly and against the National Hemophilia Foundation. The claim stems from an incident dating back to 2014. Our attorneys plan to appeal the  decision.

NHF has a bright future; we refuse to let inherited challenges overshadow the present and critical mission we are committed to each and every day.

FOR IMMEDIATE RELEASE

On Saturday, September 10, 2022, BioMarin announced that a hemophilia A patient participating in a gene therapy clinical trial – in which they had been treated with BMN270 – has been diagnosed with pre B cell acute lymphoblastic leukemia. 

The National Hemophilia Foundation (NHF) and Medscape are pleased to announce the first live activity in a Virtual Grand Round Series on “Gene Therapy for Patients with Hemophilia.”

The U.S. Food and Drug Administration has accepted for priority review the Biologics License Application (BLA) for Sanofi’s investigational therapy efanesoctocog alfa (BIVV001).

A newly released survey of individuals with Hereditary Factor X Deficiency (HFXD) and caregivers of those with HFXD yielded noteworthy insights related to treatment and quality of life (QoL).

Get the latest news from the 2022 Bleeding Disorders Conference with the Wrap-up issue of the NHF Show Daily!



Read it now

Get the latest news from the 2022 Bleeding Disorders Conference with the Saturday issue of the NHF Show Daily!



Read it now

Get the latest news from the 2022 Bleeding Disorders Conference with the Saturday issue of the NHF Show Daily!



Read it now

Get the latest news from the 2022 Bleeding Disorders Conference with Friday issue of the NHF Show Daily!



Read it now

The European Commission granted conditional approval to BioMarin's Hemophilia A gene therapy Valoctocogene Roxaparvovec which will be known as Valrox.

With this approval in Europe the regulators will monitor the use of Valrox for 15 years to ensure safety of the therapy.

Click here to the BioMarin press release.

The 2022 Bleeding Disorders Conference has kicked off with first issue of the NHF Show Daily! Read it now.

The Plasma Protein Therapeutics Association

FOR IMMEDIATE RELEASE   

MEDIA CONTACT                                                                            

Ilana Ostrin   

Senior Director of Public Relations and Communications  

iostrin@hemophilia.org

212-328-3769 

Aug. 28, 2022

BioMarin recently shared findings from a phase 3 clinical trial (GENEr8-1) evaluating the presence of vector DNA in patients two years after they received a single dose of valoctocogene roxaparvovec, the company’s investigational hemophilia A gene therapy.

Novo Nordisk recently announced that the U.S.

As investigational hemophilia gene therapies move closer to regulatory authorization, community stakeholders have recognized the acute need for people with hemophilia (PWH) and healthcare professionals (HCPs) to be fully engaged in shared decision making (SDM).

Sanofi recently shared new clinical trial updates for their investigational hemophilia therapy fitusiran. The Phase 3 ATLAS-PPX study is evaluating the efficacy and safety of fitusiran (80 mg) when administered prophylactically (once per month) to adults and adolescents with severe hemophilia A or B. The 80 participants enrolled in the trial, all of whom are 12 years of age and up, had been previously treated with existing factor replacement therapy or bypassing agent (BPA) prophylaxis.

The recent Congress of the International Society on Thrombosis and Haemostasis (ISTH) included some noteworthy findings on the rare blood disorders news front. Hemab Therapeutics, which specializes in developing therapies that target serious underserved bleeding and thrombotic disorders, presented new preclinical data on HMB-001.

Sanofi and Swedish Orphan Biovitrum AB (Sobi®) recently shared positive results from the XTEND-1 pivotal phase 3 study of efanesoctocog alfa (BIVV001).

BIVV001 is an investigational recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic intravenous infusions. The results were presented at the 2022 International Society on Thrombosis and Haemostasis Congress, held July 9-13th in London.

FOR IMMEDIATE RELEASE



Abortion and contraception can both be critical components of an individual’s health care journey. However, it is important to not conflate abortion and birth control as one in the same – both are medical in nature but serve different purposes.



Yet, many policymakers are treating abortion and contraception as one-in-the-same, and as something that must be controlled or limited.

Meet NHF's Chapter Development Specialist, Nick Kallinicou! Nick took is passion for meaningful and impactful work and joined NHF. He continues to be a vital part of the NHF community and assisting families and patients. Learn more about his role at NHF!

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

My name is Nick Kallinicou, and I am the Chapter Development Specialist on the Chapter Services team.

 

NHF’s Medical and Scientific Advisory Council (MASAC) has approved a new recommendation on genotyping for people with hemophilia, which were endorsed by the NHF Board of Directors on July 6th.

 

FOR IMMEDIATE RELEASE

MEDIA CONTACT:


Ilana Ostrin

212 328 3769



Patients, families and caregivers reunite at the 2022 Bleeding Disorders Conference

FOR IMMEDIATE RELEASE

July 8, 2022

 

FOR IMMEDIATE RELEASE



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.

FOR IMMEDIATE RELEASE

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.

Resource Links

Hawaii Chapter - NHF
75-5660 Kopiko Street, Suite C7 #294
Kailua-Kona, HI 96740
Phone 808-284-7417

© Hawaii Chapter Hemophilia Foundation 2022

Crafted by Firespring