Rhythm and Que's
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Lenexa, Kansas
FDA Approves Qfitlia: New Hemophilia Therapy Breakthrough
FDA Approves Qfitlia: New Hemophilia Therapy Breakthrough
Qfitlia™ is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors.
Paper Finds Bone Health Screening Rates at HTCs Represent Clinical Care Gap
Paper Finds Bone Health Screening Rates at HTCs Represent Clinical Care Gap
A newly published journal article examined current practices of HTCs for screening and assessing bone health in people with hemophilia and von Willebrand disease.
Breaking News: Takeda Announces Discontinuation of Two Hemophilia Treatments
Breaking News: Takeda Announces Discontinuation of Two Hemophilia Treatments
Takeda Pharmaceuticals announced today it will globally discontinue two of its hemophilia treatments: HEMOFIL® M [Antihemophilic Factor (Human), Method M, Monoclonal Purified] and RECOMBINATE® [Antihemophilic Factor (Recombinant)].
Pfizer Discontinues Beqvez Hemophilia B Gene Therapy
Pfizer Discontinues Beqvez Hemophilia B Gene Therapy
Pfizer stops global development and commercialization of their hemophilia B gene therapy.
Clinical Practice and Hemophilia Gene Therapy the Focus of New Accredited Activity
Clinical Practice and Hemophilia Gene Therapy the Focus of New Accredited Activity
This activity is intended for learners to enhance their ability to recognize the key differences in gene therapies for hemophilia A compared with hemophilia B, and to leverage evolving data about gene therapies for hemophilia to better care for patients.
FDA Approves Subcutaneous Therapy for Hemophilia Patients with Inhibitors
FDA Approves Subcutaneous Therapy for Hemophilia Patients with Inhibitors
Alhemo is FDA approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.
Research Opportunity: Michigan Study Explores Gene Therapy Understanding in Hemophilia Community
Research Opportunity: Michigan Study Explores Gene Therapy Understanding in Hemophilia Community
The National Bleeding Disorders Foundation is proud to support an important research initiative led by Emily Agen, a genetic counseling graduate student at the University of Michigan, who is
Trial Updates for Investigational Hemophilia A Gene Therapy Shared at ASH
Trial Updates for Investigational Hemophilia A Gene Therapy Shared at ASH
Expression Therapeutics investigational hemophilia A gene therapy is developed with novel lentiviral vectors.
HTC Looks at Family Pedigrees and Hemophilia Carrier Screening
HTC Looks at Family Pedigrees and Hemophilia Carrier Screening
Female hemophilia carriers often go undiagnosed. Learn about the importance of identifying and supporting these individuals to ensure equitable access to care.
Provide Your Feedback on the WFH Hemophilia Guidelines Today!
Provide Your Feedback on the WFH Hemophilia Guidelines Today!
The WFH seeks feedback on its Hemophilia Management Guidelines. Help shape the future of these critical resources by taking a short survey. Your input matters!
All About HTCs
In this video, you will hear about how to prepare for a visit to your HTC, or Hemophilia Treatment Clinic. You will also learn a bit about the different healthcare providers available to you at an HTC.
Communicating with a Hematologist
In this video, you will hear about how to prepare for a visit with your hematologist and how to advocate for your needs to get the best possible care.
Communicating with an OB/GYN
In this video, you will hear about how to prepare for a visit with your gynecologist and how to advocate for your needs to get the best possible care.
Communicating with a Healthcare Provider
In this video, you will hear about how to prepare for a visit with your healthcare provider and how to advocate for your needs to get the best possible care.
Todo Sobre los HTC
En este vídeo, le explicaremos cómo prepararse para una visita a su HTC, o Clínica de Tratamiento de la Hemofilia. También aprenderá un poco acerca de los diferentes proveedores de atención médica disponibles para usted en un HTC.
Hablando con un Hematólogo
En este vídeo, le explicaremos cómo prepararse para una visita con su hematólogo y cómo defender sus necesidades para obtener la mejor atención posible.
Hablando con un Ginecólogo Obstetra
En este vídeo, aprenderás a prepararte para una visita al ginecólogo y a defender tus necesidades para recibir la mejor atención posible.
Hablando con un Profesional Médico
En este vídeo le explicamos cómo prepararse para una visita al médico y cómo defender sus necesidades para recibir la mejor atención posible.
Consejos para Traducción e Interpretación
En este vídeo, aprenderás un poco más sobre betteryouknow.org, concretamente sobre los distintos recursos imprimibles disponibles en el sitio web y cómo utilizarlos mejor.
Recursos Para Usted
¿Cómo puede betteryouknow.org satisfacer mejor sus necesidades? En este vídeo le mostraremos este sitio web y le indicaremos qué páginas pueden resultarle más útiles en los distintos momentos de su diagnóstico.
Sangrando Qué
¿Qué son los trastornos hemorrágicos? En este vídeo aprenderás qué son los trastornos hemorrágicos, sus signos y síntomas, y por qué es importante buscar atención médica.
Lo Que Viene Despues
Tiene signos y síntomas de un trastorno hemorrágico, ¿y ahora qué? En este vídeo, le explicaremos los siguientes pasos para buscar un diagnóstico de trastorno hemorrágico que le ayude a recibir la atención que necesita.
More From Betteryouknow.org
In this video, you will learn a bit more about betteryouknow.org, specifically more about the various printable resources available on the website and how to best use them.
What Betteryouknow.org Has for You
How can betteryouknow.org best serve your needs? In this video, you’ll get a tour through this website, with pointers on which webpages can be most helpful throughout the different points in your diagnostic journey.
Bleeding What?
What even are bleeding disorders? In this video, you’ll learn about what bleeding disorders are, their signs and symptoms, and why it’s important to seek out care.
What's Next?
You have signs and symptoms of a bleeding disorder – what now? In this video, we will walk through the next steps in seeking out a bleeding disorder diagnosis to ultimately help you receive the care you need.
Bleeding Disorders Conference
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Aurora, Colorado
FDA Approves Pfizer's HYMPAVZI for Hemophilia A and B Treatment
FDA Approves Pfizer's HYMPAVZI for Hemophilia A and B Treatment
On Oct 11, 2024, Pfizer's HYMPAVZI™ was FDA-approved for preventing bleeding episodes in hemophilia A and B, marking a major advancement in hemophilia care.
CHOP Researchers Investigate Durability of Hemophilia A Gene Therapy
CHOP Researchers Investigate Durability of Hemophilia A Gene Therapy
A new paper looked at the long-term durability of hemophilia A gene therapy.
WFH Unveils 2025 World Hemophilia Day Theme: "Access for All: Women and Girls Bleed Too"
WFH Unveils 2025 World Hemophilia Day Theme: "Access for All: Women and Girls Bleed Too"
World Hemophilia Day 2025 focuses on equal access to care for women and girls with bleeding disorders.
Enduring Education Focuses on Hemophilia Non-Factor Replacement Therapy
Enduring Education Focuses on Hemophilia Non-Factor Replacement Therapy
"Exploring Non-Factor Replacement Therapies for Hemophilia Care: Achieving Positive Outcomes," is intended for U.S.-based hematologist/oncologists, pediatricians, primary care physicians, emergency medicine physicians, nurses, pharmacists, and other members of the comprehensive hemophilia care team
BioMarin to Narrow Its Commercial Focus for Hemophilia A Gene Therapy
BioMarin to Narrow Its Commercial Focus for Hemophilia A Gene Therapy
In a new press release the company signaled their plan to limit commercial operations to the three countries where ROCTAVIAN is both approved and reimbursed as a treatment for people with hemophilia.
Attention Clinicians! New Bleeding Disorders Education Available from IHS
Attention Clinicians! New Bleeding Disorders Education Available from IHS
These new medical-based webinars will speak to key topics relevant to the care of patients with inherited bleeding disorders.
Pfizer Announces New Data from Hemophilia A Gene Therapy Study
Pfizer Announces New Data from Hemophilia A Gene Therapy Study
This clinical trial program is evaluating the efficacy and safety of a single intravenous infusion of giroctocogene fitelparvovec in more than 60 adult participants with moderately severe to severe hemophilia A.
Elevating Research with Community Voices: How Lived Experiences Drive Progress in Bleeding Disorders
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Pfizer Presents Marstacimab Trial Updates at EHA Annual Meeting
Pfizer Presents Marstacimab Trial Updates at EHA Annual Meeting
Pfizer recently provided new clinical trial updates for their investigational therapy marstacimab.
Study Provides New Data on Neurodevelopment in Younger Hemophilia Population
Study Provides New Data on Neurodevelopment in Younger Hemophilia Population
Study investigators assessed the impacts of hemophilia type and severity, intracerebral hemorrhage, hemophilia treatment type, socioeconomic status, and neurodevelopmental disorders or symptoms on cognitive, adaptive, and social-emotional functioning compared with the general population.
Explore Videos on Clinical Issues in Novel Treatment Modalities
Explore Videos on Clinical Issues in Novel Treatment Modalities
This series focused on clinical issues relevant to non-factor products and emerging novel therapies still in development for the treatment of hemophilia.
Bispecific Antibody
Discover the science behind bispecific antibodies with our engaging 3D video.
Study Suggests Prophylaxis Can Mitigate Intracranial Hemorrhage Risk
Study Suggests Prophylaxis Can Mitigate Intracranial Hemorrhage Risk
Authors of the study used retrospective data from the ATHNdataset to inform their research into intracranial hemorrhage risk in people with hemophilia A.
FDA Approval Includes Full ALTUVIIIO™ Pediatric Trial Results
FDA Approval Includes Full ALTUVIIIO™ Pediatric Trial Results
ALTUVIIIO is a recombinant factor VIII replacement therapy (rFVIII), designed to extend the half-life of the rFVIII, allowing for more sustained protection from bleeds per infusion.
Empower Your Journey: Explore the Own Your Path and THRIVE Programs with NBDF's Education Department
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FDA Approves Second Hemophilia B Gene Therapy Product
FDA Approves Second Hemophilia B Gene Therapy Product
This hemophilia B gene therapy is administered as a one-time intravenous infusion to stimulate the long-term production of clotting factor IX and prevent bleeding.
Hemophilia B Gene Therapy Trial Data Published
Hemophilia B Gene Therapy Trial Data Published
Hemgenix is currently the only hemophilia B gene therapy approved by the FDA.
Learn More About 2024 Bleeding Disorders Scholarships
Learn More About 2024 Bleeding Disorders Scholarships
The HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding disorders community!
Rebinyn Supplies Restored in 3,000 IU Vial Size
Rebinyn Supplies Restored in 3,000 IU Vial Size
Rebinyn is a recombinant factor IX extended half-life therapy for the treatment of hemophilia B.
Hemophilia B Therapy FDA Approved for Expanded Indication
Hemophilia B Therapy FDA Approved for Expanded Indication
The new indication is based on a phase 3/4 clinical trial in of patients under 12 years of age with severe or moderately severe hemophilia B.
New Hemophilia Fact Sheet Now Available
New Hemophilia Fact Sheet Now Available
The fact sheet includes basic information about hemophilia.
Novo Nordisk Experiencing Shortage of Rebinyn 3,000 IU Vials
Novo Nordisk Experiencing Shortage of Rebinyn 3,000 IU Vials
Rebinyn is a recombinant factor IX extended half-life therapy for the treatment of individuals with hemophilia B.
Study Suggests Hemophilia Patients' Preferences Evolving Along with Treatments
Study Suggests Hemophilia Patients' Preferences Evolving Along with Treatments
Study authors looked at hemophilia treatment preferences in light of less burdensome novel therapies.
Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial
Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial
Roctavian™ was approved in the summer of 2023 for the treatment of adults with severe hemophilia A
NBDF and HFA Address Concerns on WHO's Recommendation of Cryoprecipitate
NBDF and HFA Address Concerns on WHO's Recommendation of Cryoprecipitate
Organizations call on WHO to include FVIII concentrates on the EML outside of the complimentary list.
FDA Accepts Biologics License Application for Marstacimab
FDA Accepts Biologics License Application for Marstacimab
The BLA submission is supported by positive safety and efficacy data from an ongoing global phase 3 clinical trial known as BASIS
CSL Presents Three Year Treatment Data for Hemophilia B Gene Therapy
CSL Presents Three Year Treatment Data for Hemophilia B Gene Therapy
The data were presented at the recent 65th American Society of Hematology (ASH) Annual Meeting and Exposition.
Gene Therapy for Hemophilia: Progress and Conundrums
BDC Education Series for Providers: Gene Therapy for Hemophilia: Progress and Conundrums
Impact of Gene Therapy for Hemophilia on Care Delivery in the Hemophilia Treatment Center
BDC Education Series for Providers: Impact of Gene Therapy for Hemophilia on Care Delivery in the Hemophilia Treatment Center
The Science Required to Cure Hemophilia
BDC Education Series for Providers: The Science Required to Cure Hemophilia
Novo Seven Unavailable in 8 mg Vials Due to Supply Constraints Beginning in January 2024
Novo Seven Unavailable in 8 mg Vials Due to Supply Constraints Beginning in January 2024
Due to supply constraints, Novo Nordisk will be unable to provide the 8 mg vial of NovoSeven® RT from January until mid-2024. Importantly, the overall supply of NovoSeven® RT is expected to remain intact as other vial sizes are available.
First Patient Dosed with Hemophilia Gene Therapy Candidate ASC618
First Patient Dosed with Hemophilia Gene Therapy Candidate ASC618
Investigators of the phase I/II trial are evaluating safety, tolerability, and preliminary efficacy of ASC618.
Sernova Receives Dual FDA Designations for Investigational Hemophilia A Therapy
Sernova Receives Dual FDA Designations for Investigational Hemophilia A Therapy
The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process.
Study Suggests Racial and Ethnic Disparities in Hemophilia and ITI
Study Suggests Racial and Ethnic Disparities in Hemophilia and ITI
Findings of a new study have uncovered specific disparities in hemophilia care by focusing on the use of immune tolerance therapy (ITI) in people with hemophilia A and an inhibitor.
New Hemophilia Gene Therapy Video Presentations Available
New Hemophilia Gene Therapy Video Presentations Available
These talks were originally delivered live and in-person as part of NBDF’s 75th Bleeding Disorders Conference.
Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations
Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations
Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on treatment, research, and other general health concerns of the inheritable bleeding disorders community.
International Hemophilia Survey Suggests Psychosocial Challenges and Lack of Shared Decision Making
International Hemophilia Survey Suggests Psychosocial Challenges and Lack of Shared Decision Making
The survey included more than 2,700 participants representing four continents and 11 countries.
Investigational T-Cell Therapy for Hemophilia A Inhibitors Receives Orphan Drug Designation
Investigational T-Cell Therapy for Hemophilia A Inhibitors Receives Orphan Drug Designation
Orphan drug designation is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States.
New Study Looks at Hemophilia Survival Rate Disparities
New Study Looks at Hemophilia Survival Rate Disparities
Black men experience much lower hemophilia survival rates compared to their white counterparts.
Patient Safety in the Age of Gene Therapy a Central Focus of Review Article
Patient Safety in the Age of Gene Therapy a Central Focus of Review Article
This paper takes a broad, holistic view of patient safety in the era of hemophilia gene therapy.
New Trial Data Shows ALTUVIIIO™ Prophylaxis Effective in Children
New Trial Data Shows ALTUVIIIO™ Prophylaxis Effective in Children
Results from the Phase 3 XTEND-Kids study were presented during a late-breaking session at the June Annual Meeting of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal.
Hemophilia A Gene Therapy Product Receives U.S. FDA Approval
Hemophilia A Gene Therapy Product Receives U.S. FDA Approval
Discover ROCTAVIAN™, an FDA-approved gene therapy for hemophilia A. Explore its efficacy and potential to transform treatment.
Patient-Clinician Partnerships the Theme of ASCO Presidential Address
Patient-Clinician Partnerships the Theme of ASCO Presidential Address
Discover insights on healthcare partnerships from ASCO President Eric P. Winer, both as an oncologist and patient.
First Hemophilia B Patient Dosed with Gene Therapy Product
First Hemophilia B Patient Dosed with Gene Therapy Product
CSL Behring announces the first HEMGENIX gene therapy dosing for hemophilia B. Learn more about this milestone and its indications.
Gene Therapy as a Novel Approach to Achieving ITI in FVIII Inhibitors Reviewed
Gene Therapy as a Novel Approach to Achieving ITI in FVIII Inhibitors Reviewed
Explore the potential of gene therapy for hemophilia A with inhibitors. Learn about challenges, current strategies, and its impact on patients.
Pfizer Highlights Phase 3 Trial Results of Marstacimab
Pfizer Highlights Phase 3 Trial Results of Marstacimab
Explore the positive phase 3 trial results for Pfizer's marstacimab, a subcutaneous therapy for hemophilia A and B patients with or without inhibitors.
It is Family: A Conversation with Saylor Behrens
It is Family: A Conversation with Saylor Behrens
Saylor Behrens discusses hemophilia awareness, family, and more. Read the interview with Saylor, whose brother has hemophilia A.
Updates on Generic Desmopressin Product
Updates on Generic Desmopressin Product
Stay informed about the availability and insights on cost, access, and challenges related to the generic desmopressin-based nasal spray. Learn more at NBDF.
MLOF Genotyping Project Informed Inhibitor Risk
MLOF Genotyping Project Informed Inhibitor Risk
Learn about the MLOF Genotyping Project and its insights into inhibitor risk among individuals with hemophilia A. Genetic variations can inform future plans.
FDA Requests Additional Information on Concizumab from Novo Nordisk
FDA Requests Additional Information on Concizumab from Novo Nordisk
Novo Nordisk's concizumab faces delays as the FDA requests more information. Get updates on the status of this subcutaneous therapy for hemophilia A and B.
Sanofi Announces Publication of Two Phase 3 Fitusiran Trials
Sanofi Announces Publication of Two Phase 3 Fitusiran Trials
Sanofi's phase 3 trials of Fitusiran bring hope for improved hemophilia treatment. Explore the breakthroughs in this investigational therapy.
Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage
Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage
Akorn Pharmaceuticals' bankruptcy won't cause a shortage. Discover its role in treating bleeding disorders and its ongoing production status.
Guilt in Mothers of Children with Hemophilia Focus of New Study
Guilt in Mothers of Children with Hemophilia Focus of New Study
Explore a recent study on guilt in mothers of children with hemophilia. Discover contributing factors, coping strategies, and more.
BioMarin Announces FDA’s Extended Review of Investigational Hemophilia Gene Therapy
BioMarin Announces FDA’s Extended Review of Investigational Hemophilia Gene Therapy
The FDA extends the review of BioMarin's ROCTAVIAN gene therapy for hemophilia A. Learn about the latest developments.
Start the Conversation: Dr. Amy Dunn on Gene Therapy
Start the Conversation: Dr. Amy Dunn on Gene Therapy
Discover the latest in hemophilia gene therapy with Dr. Amy Dunn in a conversation hosted by NHF CEO Dr. Len Valentino showing NHF's role in finding cures.
Start the Conversation: Gene Therapy – An Oasis or Mirage?
Start the Conversation: Gene Therapy – An Oasis or Mirage?
Join the conversation on gene therapy for hemophilia B with Ray Stanhope and NHF CEO Dr. Len Valentino. Explore eligibility, pricing, and access issues.
New Medscape Activity to Help Institutions Offer Gene Therapy
New Medscape Activity to Help Institutions Offer Gene Therapy
Discover NHF's accredited educational activity with Medscape, aiding healthcare providers in integrating gene therapy into collaborative care for hemophilia.
uniQure Announces Published Trial Results for Approved Gene Therapy
uniQure Announces Published Trial Results for Approved Gene Therapy
Learn about Hemgenix® gene therapy's success in reducing bleeding episodes for hemophilia B patients with published NEJM results.
FDA Approves ALTUVIIIO™ for Hemophilia A Patients
FDA Approves ALTUVIIIO™ for Hemophilia A Patients
Learn about the FDA approval of ALTUVIIIO™ for hemophilia A patients, offering sustained bleed protection and a single weekly dose.
Kopenitz family commits substantial funding to NHF health equity initiatives
Kopenitz family commits substantial funding to NHF health equity initiatives
Steve and Sheila Kopenitz support NHF's health equity initiatives for families with blood disorders. Learn about their contribution.
WFH Gene Therapy Registry Goes Live
WFH Gene Therapy Registry Goes Live
Discover the new WFH Gene Therapy Registry (GTR) for global hemophilia gene therapy safety and efficacy monitoring.
Sanofi Announces New Efanesoctocog alfa Data Published in the NEJM
Sanofi Announces New Efanesoctocog alfa Data Published in the NEJM
Sanofi's efanesoctocog alfa, a potential hemophilia A treatment, shows promise in NEJM-published clinical trial data. Learn more about this innovative therapy.
New FIX Chromogenic Assay Launched by Precision BioLogic
New FIX Chromogenic Assay Launched by Precision BioLogic
Precision BioLogic introduces an FDA-cleared FIX Chromogenic Assay for hemophilia B patients aged two and above. Learn more.
Biomarin Provides Hemophilia A Clinical Development Program Updates
Biomarin Provides Hemophilia A Clinical Development Program Updates
Stay informed about BioMarin's latest updates on valoctocogene roxaparvovec's clinical development for Hemophilia A.
Pfizer Announces Trial Updates for Investigational Hemophilia Gene Therapy
Pfizer Announces Trial Updates for Investigational Hemophilia Gene Therapy
Pfizer reports promising results from a gene therapy trial for hemophilia B, showing significant reductions in bleeding rates.
NHF's Own Your Path program begins recruitment
NHF's Own Your Path program begins recruitment
Recruitment is now open for Own Your Path- a program for young adult males with hemophilia who are currently in the transition years between 18-29. We know that maintaining adherence to a prophy regimen can have short and long -term health benefits.
Spark Announces SPK-8011 Updates at ASH
Spark Announces SPK-8011 Updates at ASH
The updates were presented at the 64th American Society of Hematology (ASH) annual meeting in New Orleans, Louisiana
First Hemophilia B Gene Therapy Approved by FDA
First Hemophilia B Gene Therapy Approved by FDA
On November 22, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based gene therapy.
Meet Former JGP Fellow, Dr. Lacramioara Ivanciu!
Meet Former JGP Fellow, Dr. Lacramioara Ivanciu!
In 2010, as a postdoctoral fellow in Dr. Rodney Camire’s laboratory, she was awarded NHF’s Judith Graham Pool Postdoctoral Research Fellowship.
Meet Former JGP Fellow, Dr. Jyoti Mathur!
Meet Former JGP Fellow, Dr. Jyoti Mathur!
From 2007 to 2009, Dr. Mathur was a fellow with the Judith Graham Pool Postdoctoral Research Fellowship.
Meet Former JGP Fellow, Dr. Seema Patel!
Meet Former JGP Fellow, Dr. Seema Patel!
Dr. Semma Patel is an assistant professor at Emory University who became a JGP fellow in 2019.
Meet JGP Fellow, Dr. Sean Quinn!
Meet JGP Fellow, Dr. Sean Quinn!
Dr. Quinn became a JGP fellow in 2021. Currently, he is a postdoctoral research fellow at the Children’s Hospital of Philadelphia.
Meet JGP Fellow, Dr. Kaushik Das!
Meet JGP Fellow, Dr. Kaushik Das!
Dr. Kaushik Das is a postdoctoral research associate at the University of Texas Health Science Center at Tyler. He joined the JGP Fellowship in 2022.
Meet Former JGP Fellow, Dr. Xuejie Chen!
Meet Former JGP Fellow, Dr. Xuejie Chen!
Before becoming a research associate at the University of North Carolina at Chapel Hill, Dr. Chen was a postdoctoral research fellow in laboratory of Dr. Darrel Stafford. In 2020, she received a JGP fellowship to pursue research in the field of hematology.
Meet JGP Fellow, Dr. Vishal Srivastava!
Meet JGP Fellow, Dr. Vishal Srivastava!
Dr. Srivastava is a JGP fellow working in Dr. Bin Zhang's lab at the Genomic Medicine Institute at the Cleveland Clinic.
Meet Former JGP Fellow, Dr. Elizabeth Chappell, MBA!
Meet Former JGP Fellow, Dr. Elizabeth Chappell, MBA!
Dr. Chappell is a senior consultant in analytical services at BioTechLogic, Inc. She earned her PhD in Pharmaceutical Sciences from UNC Chapel Hill in 2013.
BioMarin Shares Community Update for Valoctocogene Roxaparvovec
BioMarin Shares Community Update for Valoctocogene Roxaparvovec
Valoctocogene roxaparvovec uses adeno-associated viruses (AAVs) vectors to deliver the DNA genetic codes that illicit the production of the factor VIII in people with hemophilia A.
Recruitment Reopened for Phase 3 AFFINE Gene Therapy Trial
Recruitment Reopened for Phase 3 AFFINE Gene Therapy Trial
This trial is evaluating the efficacy and safety of a single infusion of an investigational gene therapy in individuals with moderately severe to severe hemophilia A
Virtual Grand Round Series to Focus on Hemophilia Gene Therapy
Virtual Grand Round Series to Focus on Hemophilia Gene Therapy
This program is presented through a collaboration between the NHF and Medscape.
FDA Grants Priority Review for Sanofi’s BIVV001
FDA Grants Priority Review for Sanofi’s BIVV001
BIVV001 is a recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic intravenous infusions.
BioMarin Receives Conditional Approval in Europe for Gene Therapy
BioMarin Receives Conditional Approval in Europe for Gene Therapy
The European Commission granted conditional approval to BioMarin's Hemophilia A gene therapy Valoctocogene Roxaparvovec which will be known as Roctavian.
BioMarin Presents Abstract on Vector DNA Transmission Risk at ISTH
BioMarin Presents Abstract on Vector DNA Transmission Risk at ISTH
The focus of the abstract is the investigational hemophilia A gene therapy, valoctocogene roxaparvovec.
New Prophylaxis Approval for REBINYN®
New Prophylaxis Approval for REBINYN®
REBINYN® first received FDA approval in May of 2017.
Paper Features Q&A Resource on Shared Decision Making and Gene Therapy
Paper Features Q&A Resource on Shared Decision Making and Gene Therapy
The Q&A resource highlighted here is meant to encompass all stages of a patient's decision making journey in gene therapy.
Sanofi Provides Fitusiran Trial Updates at ISTH
Sanofi Provides Fitusiran Trial Updates at ISTH
These updates were presented during the International Society on Thrombosis and Haemostasis (ISTH) Congress in London.
Sanofi and Sobi® Report Progress with BIVV001 at International Congress
Sanofi and Sobi® Report Progress with BIVV001 at International Congress
BIVV001 is an investigational recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in people with hemophilia A.
New Genotyping Recommendations Issued by MASAC
New Genotyping Recommendations Issued by MASAC
The new recommendation describes the proven benefits of genotyping people with hemophilia.
National Hemophilia Foundation Requests Risk Evaluation and Mitigation Strategy for Pending Gene Therapy Treatments
National Hemophilia Foundation Requests Risk Evaluation and Mitigation Strategy for Pending Gene Therapy Treatments
FOR IMMEDIATE RELEASE
July 8, 2022
Diabetes Risk in Hemophilia vs Unaffected Groups Compared in Retrospective Study
Diabetes Risk in Hemophilia vs Unaffected Groups Compared in Retrospective Study
The hemophilia data for this study was drawn from deidentified medical records from four North American hemophilia treatment centers.
Bayer Announces Discontinuation of Kogenate® FS
Bayer Announces Discontinuation of Kogenate® FS
Kogenate® was first approved by the U.S. Food and Drug Administration in 1993.
FDA Grants Breakthrough Status to BIVV001
FDA Grants Breakthrough Status to 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 infusions.
BioMarin Announces Delay in Application to FDA
BioMarin Announces Delay in Application to FDA
Roctavian is Biomarin’s investigational hemophilia A gene therapy, which is currently in multiple ongoing clinical trials
FDA Accepts Priority Review BLA for Hemophilia B Gene Therapy
FDA Accepts Priority Review BLA for Hemophilia B Gene Therapy
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.
Bone Health in VWD and Hemophilia Carriers the Focus of Retrospective Study
Bone Health in VWD and Hemophilia Carriers the Focus of Retrospective Study
The study looked at the prevalence and risk factors of osteoporosis, osteoarthritis, and bone fractures.
New Data Shows Eptacog Beta Safe and Effective in Pediatric Patients
New Data Shows Eptacog Beta Safe and Effective in Pediatric Patients
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.
NHLBI Grant Supports Scientific Research to Enhance Hemophilia A Gene Therapies
NHLBI Grant Supports Scientific Research to Enhance Hemophilia A Gene Therapies
The new grant will fund basic and translational studies to improve gene therapies for hemophilia A.
NHF Statement on World Hemophilia Day
NHF Statement on World Hemophilia Day
FOR IMMEDIATE RELEASE
Media Contact:
Ilana Ostrin
iostrin@hemophilia.org
212-328-3769
April 17, 2022
Hemophilia in the Romanov Family
Hemophilia in the Romanov Family
Insights from historian Dr. Helen Rappaport
First Patient Dosed in Freeline’s Dose-Confirmation Trial for Hemophilia B Gene Therapy
First Patient Dosed in Freeline’s Dose-Confirmation Trial for Hemophilia B Gene Therapy
The company's investigational gene therapy has been developed for individuals with severe or moderately severe hemophilia B.
Starting the Conversation with Dr. Johnny Mahlangu
Starting the Conversation with Dr. Johnny Mahlangu
Dr. Johnny Mahlangu graciously 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!
Candid Conversations: What Do the Docs Think
NBDF provides free quality education on issues that matter to the bleeding disorders community. This video is on candid conversations, what do the docs think?
Advancements in Treatment for Hemophilia
NBDF provides free quality education on issues that matter to the bleeding disorders community. This video is on the advancements in treatment for hemophilia
Gene Therapy: Where Do We Go From Here?
NBDF provides free quality education on issues that matter to the bleeding disorders community. This video is on gene therapy: where do we go from here
Basics of Hemophilia
NBDF provides free quality education on issues that matter to the bleeding disorders community. This video is on the basics of hemophilia
New Medscape Activity Seeks to Prepare Clinicians for Gene Therapy
New Medscape Activity Seeks to Prepare Clinicians for Gene Therapy
This program is intended for an international audience of hematologists/oncologists, pediatricians, hematology/oncology nurses, nurse practitioners, and physician assistants.
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
Valoctocogene roxaparvovec gene therapy is being investigated for safety and efficacy in adults with severe hemophilia A via multiple clinical trials.
New Study Looks at Physical Activity Risk in Patients with Hemophilia A
New Study Looks at Physical Activity Risk in Patients with Hemophilia A
This study designed to assess the link between physical activity levels, FVIII infusion, and occurrence of bleeding episodes.
Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks
Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks
The phase 1/2 trial of SIG-001 for hemophilia A has been on an FDA clinical hold since July 2021.
uniQure and CSL Behring Announce Trial Updates for Hemophilia B Gene Therapy
uniQure and CSL Behring Announce Trial Updates for Hemophilia B Gene Therapy
The HOPE-B trial is investigating etranacogene dezaparvovec in patients with severe and moderately severe hemophilia B.
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
The FDA placed a clinical hold on the company's investigational therapy in July of 2021.
Spark Announces Multiyear Data Update for Investigational Gene Therapy
Spark Announces Multiyear Data Update for Investigational Gene Therapy
This data was drawn from a phase 1/2 clinical trial of SPK-8011, Spark's investigational gene therapy for hemophilia A.
New Paper Highlights Integrated Care Model and the National HTC Network
New Paper Highlights Integrated Care Model and the National HTC Network
This paper provides a comprehensive description of the critical services and dynamic coordinating capacity of HTCs within a regional and national framework.
FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
Sangamo responds to a clinical hold.
Ameritas Awards Grant to the National Hemophilia Foundation
Ameritas Awards Grant to the National Hemophilia Foundation
Ameritas announced a $20,000 grant to the National Hemophilia Foundation (NHF) on behalf of the 2021 Rosen Award recipient, Jim Christensen, Jr., president of inSOURCE, Inc.
Patient-Reported Data Informs New Study on Sexual Health and Hemophilia
Patient-Reported Data Informs New Study on Sexual Health and Hemophilia
The data used for this study came from the Patient Reported Outcomes Burdens and Experiences (PROBE) initiative.
Q&A with My Life, Our Future Researcher: Dr. Ming Lim
Q&A with My Life, Our Future Researcher: Dr. Ming Lim
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My Life, Our Future (MLOF) is a nationwide campaign offering free genetic testing, or genotyping, for people affected by hemophilia.
Prophylaxis Indication Restored for BeneFIX®
Prophylaxis Indication Restored for BeneFIX®
BeneFIX® first received FDA approval in the United States in 1997.
FDA Places Clinical Hold on Sigilon’s Investigational Hemophilia Therapy
FDA Places Clinical Hold on Sigilon’s Investigational Hemophilia Therapy
Sigilon's cell-based therapy is designed to carry significant amounts of synthetic DNA material to elicit the production of factor VIII in individuals with hemophilia A.
Kevin Child Scholarship Deadline Set for August 2nd!
Kevin Child Scholarship Deadline Set for August 2nd!
This scholarship is awarded annually to a student with hemophilia who is about to enter or currently enrolled in a program of higher education.
Learn More About Shared Decision Making in Hemophilia
Learn More About Shared Decision Making in Hemophilia
Shared decision making (SDM), the concept by which patients
New Paper Explores Shared Decision Making in Hemophilia
New Paper Explores Shared Decision Making in Hemophilia
A new paper published in The Journal of Haemophilia Prac
Article Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy
Article Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy
In this review, the authors discuss some of the primary considerations relevant to investigational hemophilia gene therapies that employ adeno-associated viral (AAV) vectors.
BioMarin Presents Positive Safety and Efficacy Data for their Hemophilia A Gene Therapy
BioMarin Presents Positive Safety and Efficacy Data for their Hemophilia A Gene Therapy
These data were presented by BioMarin at the American Society of Gene & Cell Therapy Virtual Meeting, which was held May 11-14, 2021.
New Inhibitor Study Using MLOF Research Repository Data Published
New Inhibitor Study Using MLOF Research Repository Data Published
The study represents a significant step toward better understanding of inhibitors and improving clinical care.
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
The commercialization and licensing agreement between the two companies went into effect on May 6th.
uniQure Announces End of Clinical Hold on Hemophilia B Gene Therapy Program
uniQure Announces End of Clinical Hold on Hemophilia B Gene Therapy Program
Late last year the FDA put a clinical hold on the program in response to a single trial participant who was diagnosed with a common form of liver cancer.
uniQure Announces Findings from HCC Case Investigation
uniQure Announces Findings from HCC Case Investigation
The focus of the HOPE-B trial is etranacogene dezaparvovec, uniQure's investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.
Basics: Hemophilia
Whether you or someone in your family has been recently diagnosed with hemophilia, or you've lived with hemophilia your entire life and want a refresher
Update to Hemlibra® (emicizumab-kxwh) USPI
Update to Hemlibra® (emicizumab-kxwh) USPI
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.
FDA Partially Rescinds Added Indications for Two FIX Products
FDA Partially Rescinds Added Indications for Two FIX Products
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).
Sanofi Revises Fitusiran Dosing Regimen to Mitigate Risk of Vascular Thrombosis
Sanofi Revises Fitusiran Dosing Regimen to Mitigate Risk of Vascular Thrombosis
Evaluation of five vascular events suggests the risk of vascular thrombotic events may be greater with antithrombin (AT) levels that have been reduced below 10% of normal levels.
Spark Announces Preliminary Data from Investigational Gene Therapy Trial
Spark Announces Preliminary Data from Investigational Gene Therapy Trial
The first part of this phase 1/2 trial is designed to evaluate the safety and efficacy of SPK-8016 in adult males with clinically severe hemophilia A and no measurable inhibitor against FVIII.
Octapharma Announces Final Results of Nuwiq® Study
Octapharma Announces Final Results of Nuwiq® Study
Nuwiq® is indicated for on-demand treatment and control of bleeding episodes and routine prophylaxis to reduce the frequency of bleeding episodes and perioperative management of bleeding.
Treatment for Hemophilia: What You Need to Know
These are exciting times in hemophilia research, with many new treatment options on the horizon. Come learn about the latest results of various phases of clinical trials on gene therapy and other new treatment options to see what the future holds for the treatment of hemophilia.
BioMarin Announces Positive Phase 3 Study Data for Valoctocogene Roxaparvovec
BioMarin Announces Positive Phase 3 Study Data for Valoctocogene Roxaparvovec
The phase 3 trial currently includes 134 participants with hemophilia A, all of whom have received a single dose of the gene therapy.
Recall Notice: Voluntary Recall Mononine Coagulation Factor IX (Human)
Recall Notice: Voluntary Recall Mononine Coagulation Factor IX (Human)
The recall affects one batch of the product.
Hemo 201: What to Do When You Have No Clue
Hemophilia is a uniquely challenging and sometimes perplexing condition for physical therapists to manage and treat. Physical therapists who see patients across the lifespan may manage conditions that range from athletic injuries to frailty. Clinicians often rely on reviewing limited evidence, consulting colleagues, and ultimately critical thinking and best clinical judgment to develop a management plan. This session will outline a framework for different scenarios seen in the clinic and look at six broad management schemes.
Learning to Let Go: Fostering Independence
As parents of teens with hemophilia and an inhibitor, one of those biggest challenges, preparing to let go, can be especially difficult.
One Size Does Not Fit All: Individualized Therapy
What exactly is meant by individualized therapy? What might be the future treatment and quality-of-life implications for hemophilia patients? Learn more about this state-of-the-art approach to care, and how it could improve the health of a person living with hemophilia.
Transcription of "One Size Does Not Fit All: Individualized Therapy"
Is Hemophilia a Secret? Disclosure in a World Filled with Technology
Whether within friendships, romantic relationships, at school or in the workplace, an adult with hemophilia will confront the question of disclosing their bleeding disorder.
The Advantage of Prophylaxis for Adults with Joint Issues
It is a reality familiar to most adults with hemophilia of a certain age: repeated joint bleeds which subsequently causes joint deterioration, affecting everyday quality of life. But the story does not have to end there. Learn how prophylactic therapy can be used to halt the momentum of degenerative joint damage, decrease pain and help you regain mobility.
Transcription of "The Advantage of Prophylaxis for Adults with Joint Issues"
Forging a New Path – Gene Therapy Readiness and HTC Implementation
*CME/ACPE
Moderator/Speaker:
Steven W. Pipe, MD, Faculty Chair
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases,
Pediatric Medical Director, Hemophilia and Coagulation Disorders Program,
Director, Special Coagulation Laboratory,
University of Michigan
Ann Arbor, Michigan
Speakers:
The Next Wave: Clinical Updates on New and Emerging Treatment Options in Hemophilia (1)
*CME/ACPE
Moderator:
Stacy E. Croteau, MD, MMS
Assistant Professor of Pediatrics, Harvard Medical School Director, Hemophilia/VWD Program, Boston Children's Hospital Medical Director, Boston Hemophilia Center
Dana-Farber/Boston Children's Cancer Center & Blood Disorders Center
Boston, MA
Speakers:
The Next Wave: Clinical Updates on New and Emerging Treatment Options in Hemophilia (2)
*CME/ACPE
Moderator:
Christopher E. Walsh, MD, PhD
Associate Professor of Medicine, Division of Hematology and Medical Oncology
Icahn School of Medicine at Mount Sinai
Director, Hemophilia Treatment Center, Mount Sinai Hospital
New York, NY
Speakers:
Statement from Novo Nordisk on Rebinyn and COVID-19
Statement from Novo Nordisk on Rebinyn and COVID-19
If a hemophilia B patient on Rebinyn® requires treatment for COVID-19, Novo recommends informing the treating healthcare provider about their factor IX treatment.
Three Concizumab Trials Halted by Novo Nordisk
Three Concizumab Trials Halted by Novo Nordisk
The halted trials were investigating concizumab prophylaxis treatment in hemophilia A and B patients regardless of inhibitor status.
BioMarin Announces Three Year Updates on Investigational Gene Therapy
BioMarin Announces Three Year Updates on Investigational Gene Therapy
On December 23rd, the BioMarin submitted a Biologics License Application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration.
Shire Submits IND Application for Hemophilia A Gene Therapy Candidate
Shire Submits IND Application for Hemophilia A Gene Therapy Candidate
The therapy aims to prompt the sustained production of FVIII.
CDC Hosts Inhibitor Testing Webinar
CDC Hosts Inhibitor Testing Webinar
Join the CDC Inhibitor Testing Webinar on Thursday, July 24, at 2PM EST, to find out more about this complication.
NHF and McMaster University Collaborate on Clinical Practice Guidelines for Hemophilia Care Models
NHF and McMaster University Collaborate on Clinical Practice Guidelines for Hemophilia Care Models
The goal of this guideline is to support patient-centered clinical decision-making and optimize patient care.
New Online Resource from HOG
New Online Resource from HOG
Hemophilia of Georgia (HOG), Inc., launches “The Hemophilia, von Willebrand Disease & Platelet Disorders Handbook,” a new online resource.
Medical Advisory #415: Baxter Announces Labeling Error on Sterile Water
Medical Advisory #415: Baxter Announces Labeling Error on Sterile Water
Baxter Healthcare Corporation has sent a notice of a labeling error affecting the expiration date of the Sterile Water for Injection that is packaged as a diluent with Recombinate [Antihemophilic Factor (Recombinant)].