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If you want to be in the know about what’s happening in the bleeding disorders community, you’ve come to the right place!

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


On November 22, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based gene therapy. It is approved for the treatment of adults with hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Historically, many residential substance use disorder (SUD) facilities have denied admittance to individuals with bleeding disorders – the basis of these denials are most often the use of self-administered, intravenous medications such a as factor replacement therapies. The lack of access to residential addiction treatment facilities, can have very serious, even fatal outcomes for bleeding disorder (BD) patients in acute need of help with their addiction.

MEDIA CONTACT: 

Ilana Ostrin 

212-328-3769 
iostrin@hemophilia.org 

 

FOR IMMEDIATE RELEASE

Currently, there are over 167.5 million women (including individuals who identify as women or have the propensity to menstruate) living in the U.S. An estimated 3% of them are living with a diagnosed inheritable blood or bleeding disorder – and certainly many more are living their daily lives with pain and bleeding but are undiagnosed. Recent changes in access to women and girls’ health care are likely to have detrimental effects for the blood and bleeding disorders community.

Back Again! The Annual Red Tie Soiree Returns to Honor Community Members in San Francisco

On October 15, the Red Tie Soiree, a yearly gala hosted by the National Hemophilia Foundation, was held in-person for the first time in three years. The community event was held for the first-time on the West Coast, welcoming guests to the historic Julia Morgan Ballroom in San Francisco.

Tell us a little bit about yourself!

My name is Dr. Lacramioara Ivanciu; I’m a resident assistant professor of pediatrics in the Perelman School of Medicine at the University of Pennsylvania. In 2010, when I was a postdoctoral fellow in Dr. Rodney Camire’s laboratory, I was awarded NHF’s Judith Graham Pool Postdoctoral Research Fellowship.

Wonderful! What did your research focus on?

Tell us a little bit about yourself!

I’m Dr. Jyoti Mathur. From 2007 to 2009, I was a fellow with the Judith Graham Pool Postdoctoral Research Fellowship. When I was selected for the fellowship, I was a postdoctoral fellow at Stanford Medical School.

What did your research focus on?

Tell us a little bit about yourself!

Hi! My name is Dr. Laura Haynes, and I work at the Life Sciences Institute at the University of Michigan as a research investigator. I learned about the JGP Fellowship through mentors and colleagues and in 2018, I was granted fellowship.

What did your research focus on?

My work focused on the protein engineering of Plasminogen Activator 1 to develop novel regulators of the fibrinolytic and hemostatic pathways.

Tell us a little bit about yourself!

My name is Dr. Semma Patel. I’m an assistant professor at Emory University. I became a JGP Fellow in 2019, when I was working at Emory as an associate academic research scientist. I had learned about the program from my mentor.

What was the focus of your research?

My project investigated the epitopes recognized in the early immune response to Factor VIII.

How did the JGP Fellowship support your research at the time?

Tell us a little bit about yourself!

I’m Dr. Lisa Smith Webb and I am an associate professor of molecular biology and chemistry at Christopher Newport University. And along with being a Better You Know advocate and member of the bleeding disorder community, I’m a proud past JGP fellow! I was a fellow from 2002 to 2004.

How did you hear about the JGP Fellowship? Had you heard of Judith Graham Pool prior to the fellowship?

Tell us a little bit about yourself!

I’m Dr. Satish Nandakumar; I am an assistant professor at the Albert Einstein College of Medicine. I was awarded the Judith Graham Pool Postdoctoral Research Fellowship when I was working as a postdoctoral fellow in 2017. Before that, I completed my graduate work at St. Jude's Children's Research Hospital in Memphis, Tennessee.

How did you hear about the JGP Fellowship?

My mentor told me about the fellowship and the opportunities it could provide my work.

Tell us a little bit about yourself!

I’m Dr. Sean Quinn; I became a JGP fellow back in 2021. Currently, I’m a postdoctoral research fellow at the Children’s Hospital of Philadelphia. I heard about the JGP Fellowship from my faculty mentor and other researchers at the Children's Hospital of Philadelphia who applied for the fellowship.

What does your research focus on?

My research examines antibody-mediated FV/FVa resistance as a therapeutic approach for hemophilia.

Tell us a little bit about yourself!

Ok! My name is Dr. Kaushik Das, and I’m a postdoctoral research associate at the University of Texas Health Science Center at Tyler. I graduated from the University of Calcutta, India and completed my doctoral studies at Association for the Cultivation of Science, India. After learning about the JGP Fellowship, I became a fellow with the program in 2022.

What is the focus of your research?

Tell us a little bit about yourself!

I’m Dr. Xuejie Chen! I earned my PhD degree in cell biology from Beijing Normal University, P. R. China. Before becoming a research associate at the University of North Carolina at Chapel Hill, I was a postdoctoral research fellow in laboratory of Dr. Darrel Stafford. In 2020, I was honored to receive a JGP fellowship to pursue research in the field of hematology.

What did your research focus on?

Tell us a little bit about yourself!

I’m Dr. Vishal Srivastava, and I am a JGP fellow currently working in Dr. Bin Zhang's lab at the Genomic Medicine Institute at the Cleveland Clinic. I learned about the program through my principal investigator, who received this award previously.

What does your research focus on?

I’m studying the rescue of FVIII mutant expression by translational and post-translational modulation using small molecule therapy.

Biomarin recently provided an “Update for the Hemophilia Community,” on the ongoing clinical trial program for its investigational hemophilia A gene therapy, valoctocogene roxaparvovec.

FOR IMMEDIATE RELEASE

MEDIA CONTACT:

Ilana Ostrin

212-328-3769
iostrin@hemophilia.org

Meet NHF's Vice President of Research Strategy, Michelle Witkop! Michelle took her passion for pain management and impactful work and joined NHF. She continues to be a vital part of the NHF community and assisting families and patients. Learn more about her role at NHF and her time working with the community!

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

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

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