siRNA Treatment
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Learn About siRNA Treatment
Takeda Updates HEMOFIL® M and RECOMBINATE Availability for Hemophilia A Patients
Takeda Updates HEMOFIL® M and RECOMBINATE Availability for Hemophilia A Patients
HEMGENIX Gene Therapy Facing Temporary Global Supply Disruption
HEMGENIX Gene Therapy Facing Temporary Global Supply Disruption
What people with hemophilia B need to know.
NBDF and WFH statement on severe adverse event with marstacimab rebalancing agent for hemophilia
NBDF and WFH statement on severe adverse event with marstacimab rebalancing agent for hemophilia
The statement details reported adverse event, ongoing investigation, and plans to update the community.
Facts About Inhibitors Now Available in Arabic!
Facts About Inhibitors Now Available in Arabic!
This booklet includes information about how inhibitors develop in people with hemophilia, key risk factors, and tips on getting screened.
NBDF’s 2025 Research Roundtable: Advancing Bleeding Disorders Research for Women and Girls
NBDF’s 2025 Research Roundtable: Advancing Bleeding Disorders Research for Women and Girls
The meeting convened researchers, industry leaders, nonprofit organizations, and lived experience experts to discuss ways to enhance the inclusion of women and girls with bleeding disorders in research.
MASAC Approves Recommendations on Non-Factor Hemophilia Therapies, HTC Social Worker Roles
MASAC Approves Recommendations on Non-Factor Hemophilia Therapies, HTC Social Worker Roles
The three new documents approved by NBDF's Medical and Scientific Advisory Council include important recommendations on the off-label use of emicizumab in acquired hemophilia, the use of anti-tissue factor pathway inhibitors in hemophilia, and the role of HTC social workers.
Tratamiento anti-IVFT
El tratamiento anti-IVFT constituye una nueva opción para personas que tienen hemofilia A o B, con o sin inhibidores.
WFH Publishes First Ever Guidelines for Hemophilia AAV Gene Therapy
WFH Publishes First Ever Guidelines for Hemophilia AAV Gene Therapy
The new comprehensive and holistic guidelines offer physicians and other prescribers access to the latest evidence-based clinical guidance and help patients make more informed decisions about gene therapy.
IHS Hosts New Webinar Series to Tackle Clinical Issues in Bleeding Disorders
IHS Hosts New Webinar Series to Tackle Clinical Issues in Bleeding Disorders
The Irish Haemophilia Society is hosting a series of clinical webinars on key topics developed for healthcare providers practicing in the global bleeding disorders community.
WHO Updates Essential Medicines List to Improve Bleeding Disorders Care
WHO Updates Essential Medicines List to Improve Bleeding Disorders Care
WHO adds emicizumab and recombinant clotting factors to its Essential Medicines List, a major win for global bleeding disorders care and access.
Anti-TFPI Treatment
Discover how Anti-TFPI helps support clotting for people with hemophilia A or B in this 3D video.
New Approval Expands Indication for Recombinant VWD Therapy
New Approval Expands Indication for Recombinant VWD Therapy
FDA expands VONVENDI approval to include prophylaxis for adults with von Willebrand disease and pediatric on-demand use, advancing care across all VWD types.
Unlocking the Clotting Cascade: Hands-On Learning at BDC 2025
Unlocking the Clotting Cascade: Hands-On Learning at BDC 2025
A hands-on BDC 2025 workshop used craft supplies to make the clotting process easy to see and understand.
The Treatment Landscape: New Developments in Bleeding Disorders Care
The Treatment Landscape: New Developments in Bleeding Disorders Care
Discover new therapies for bleeding disorders, from extended half-life products to rebalancing agents and novel devices shared at BDC 2025.
FDA Approves Concizumab-mtci for Hemophilia Patients Without Inhibitors
FDA Approves Concizumab-mtci for Hemophilia Patients Without Inhibitors
Concizumab-mtci is a subcutaneous therapy, which is developed using a synthetically produced (recombinant) antibody derived from a single cell clone, designed to be equally effective in individuals with hemophilia A and B, irrespective of inhibitor status.
Literature Review Reaffirms Clinical Relevance of Von Willebrand Disease Guideline
Literature Review Reaffirms Clinical Relevance of Von Willebrand Disease Guideline
A comprehensive literature review was conducted to ensure that the von Willebrand disease guidelines were still clinically appropriate and informed by the research described in more current peer-reviewed papers.
Access New Healthcare Provider Education on Bone & Mental Health in Women with Bleeding Disorders
Access New Healthcare Provider Education on Bone & Mental Health in Women with Bleeding Disorders
This activity explores illustrative case studies, presentations, and thoughtful discussions about the critical need for regular screening for anxiety/depression and bone health in women with inherited bleeding disorders.
FDA Approved Test Measures Concizumab-mtci in Hemophilia Patients
FDA Approved Test Measures Concizumab-mtci in Hemophilia Patients
The ConcizuTrace ELISA CDx test was developed as a new option for determining the amount of concizumab-mtci in hemophilia patients.
NEJM Publishes Follow-up Data on Hemophilia B Gene Therapy Product
NEJM Publishes Follow-up Data on Hemophilia B Gene Therapy Product
A newly published paper includes safety and efficacy data in hemophilia B patients who have received BEQVEZ.
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.
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)].
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.
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!
New Study Looks at GI Bleeding in von Willebrand Disease
New Study Looks at GI Bleeding in von Willebrand Disease
The authors of this new paper conducted a retrospective medical chart review focused on GI bleeding in von Willebrand disease patients, which poses significant challenges for clinicians.
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.
New Documents Cover Bleeding Disorders, Bone Health, Mental Health Facility Access
New Documents Cover Bleeding Disorders, Bone Health, Mental Health Facility Access
The National Bleeding Disorders Foundation (NBDF) is pleased to announce that three new documents have been issued by the Medical and Scientific Advisory Council (MASAC).
Anticuerpo Biespecífico
Explora el fascinante mundo de los anticuerpos biespecíficos con este vídeo en 3D.
HANDI Highlights Resources for Historically Underserved Population
HANDI Highlights Resources for Historically Underserved Population
This HANDI Highlights is dedicated to inherited bleeding disorders, and women, girls, and people with the propensity to menstruate.
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.
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
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.
Elevating Research with Community Voices: How Lived Experiences Drive Progress in Bleeding Disorders
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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.
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.
Empower Your Journey: Explore the Own Your Path and THRIVE Programs with NBDF's Education Department
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First Sickle Cell Patient to Receive Gene Therapy Featured in NYT
First Sickle Cell Patient to Receive Gene Therapy Featured in NYT
Sickle cell disease is linked to an abnormality in the hemoglobin protein, which normally helps carry oxygen throughout the body via red blood cells. This causes red blood cells, normally disc shaped and flexible enough to travel smoothly in the body, to become rigid and misshapen to resemble a “C” or sickle.
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.
NBDF Announces New Treatment Recommendations for Bleeding Disorders
NBDF Announces New Treatment Recommendations for Bleeding Disorders
New documents issued by NBDF’s Medical and Scientific Advisory Council (MASAC) cover a wide range of topics relevant to bleeding disorder patients in the U.S. and abroad.
New Review Weighs Bleeding and Clotting Risk in ITP
New Review Weighs Bleeding and Clotting Risk in ITP
Primary immune thrombocytopenia is a rare autoimmune disorder that causes a significant reduction in platelets, which are necessary for effective coagulation.
Take the HTC Patient Satisfaction Survey
Take the HTC Patient Satisfaction Survey
PSS input from affected individuals and families help HTCs identify areas where they may have strengths and where they can improve.
Intellia Therapeutics Withdraws from Hemophilia B Gene Editing Program, Leaving Regeneron in Charge
Intellia Therapeutics Withdraws from Hemophilia B Gene Editing Program, Leaving Regeneron in Charge
In a significant development within the gene editing arena, Intellia Therapeutics has announced its decision to opt out of a hemophilia B program, relinquishing control of CRISPR/Cas9-based gene editing work on factor IX treatments to its partner, Regeneron.
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.
Terapia de reemplazo de factor
¡Descubra los tratamientos SHL y EHL en 3D! Conozca la profilaxis y el tratamiento a demanda dentro de la cascada de coagulación.
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.
La cascada de la coagulación
Explore los entresijos de la coagulación de la sangre en un cautivador vídeo en 3D, desentrañando la cascada de la coagulación y su papel en la hemostasia.
(Video) Celebrating Nurses in Bleeding Disorders Care
(Video) Celebrating Nurses in Bleeding Disorders Care
The video features interviews with nurses who for decades have cared for patients at hemophilia treatment centers across the United States.
New Training Available to Early Career Hematologists
New Training Available to Early Career Hematologists
The five-day program is designed for second-to fourth year fellows and junior physicians for up to two years post-fellowship.
Researchers Look at Impact of Factor X Deficiency on Patients/Caregivers
Researchers Look at Impact of Factor X Deficiency on Patients/Caregivers
Hereditary factor X deficiency is an ultra-rare bleeding disorder that occurs in approximately 1 in 500,000 to 1 in a million people across the globe.
Von Willebrand Emergency Care Manual Now Available in Spanish
Von Willebrand Emergency Care Manual Now Available in Spanish
The manual is meant to be a reference point for emergency department staff who are not very familiar with von Willebrand disease
FDA Approves Therapy for Rare Disorder Affecting Red Blood Cells
FDA Approves Therapy for Rare Disorder Affecting Red Blood Cells
Paroxysmal nocturnal hemoglobinuria causes a person’s immune system to attack and prematurely breakdown red blood cells and platelets.
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.
Takeda's Voluntary Withdrawal: Alert on Misprinted Labels for VONVENDI®
Takeda's Voluntary Withdrawal: Alert on Misprinted Labels for VONVENDI®
Takeda announces a voluntary withdrawal for 650 IU VONVENDI® due to misprinted labels showing incorrect expiration dates.
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
Pilot Study Explores Educational Needs of HTC Physical Therapists
Pilot Study Explores Educational Needs of HTC Physical Therapists
The new study explored how new training opportunities based on a peer-to-peer model might be received by U.S. physical therapists at hemophilia treatment centers.
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.
FDA Grants Expanded Approval to Wilate® for Prophylaxis in All Types of VWD
FDA Grants Expanded Approval to Wilate® for Prophylaxis in All Types of VWD
Octapharma’s human plasma-derived von Willebrand Factor (VWF)/Factor VIII replacement product Wilate® has been granted expanded approval for prophylaxis by the U.S. Food and Drug Administration (FDA) to include all types of von Willebrand disease (VWD).
FDA Approves Recombinant Therapy for Congenital Thrombotic Thrombocytopenic Purpura
FDA Approves Recombinant Therapy for Congenital Thrombotic Thrombocytopenic Purpura
The U.S. Food and Drug Administration (FDA) has approved a recombinant enzyme replacement therapy for people with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood disorder.
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.
Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia
Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia
While the majority of women with von Willebrand disease (VWD) will experience heavy menstrual bleeding (HMB) often associated negative impacts on overall health and quality of life, available data relevant to effective treatment is limited.
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.
Paper Highlights Treatment for Ultra-Rare Type 1 Plasminogen Deficiency
Paper Highlights Treatment for Ultra-Rare Type 1 Plasminogen Deficiency
Type 1 plasminogen deficiency, also known as hypoplasminogenemia, is a very rare genetic disorder
Factor Replacement Therapy
Discover SHL & EHL Treatments in 3D! Learn about prophylaxis and on-demand treatment within the clotting cascade.
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.
The Clotting Cascade
Explore the intricacies of blood clotting in a captivating 3D video, unraveling the clotting cascade and its role in hemostasis.
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.
Inequities in Thrombosis and Hemostasis Lab Testing the Focus of Illustrated Review
Inequities in Thrombosis and Hemostasis Lab Testing the Focus of Illustrated Review
Explore a review on biases in laboratory reference intervals for thrombosis and hemostasis, highlighting disparities and action items for health equity.
New MASAC Documents Approved on Physical Therapy, Gene Therapy Readiness, Vaccine Guidance, and Treatment
New MASAC Documents Approved on Physical Therapy, Gene Therapy Readiness, Vaccine Guidance, and Treatment
Discover the latest MASAC documents on physical therapy, gene therapy readiness, vaccines, and treatment for inheritable bleeding disorders.
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.
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.
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.
New Document to Help Residential Substance Use Disorder Placement for Individuals with Bleeding Disorders
New Document to Help Residential Substance Use Disorder Placement for Individuals with Bleeding Disorders
The document includes best practices for HTC providers in accessing residential substance use disorder treatment.
Avances en el Tratamiento
Son tiempos emocionantes en la investigación de los trastornos hemorrágicos, con nuevas opciones de tratamiento en el horizonte.
Technologies and Advancements in Inhibitor Treatments
Treating hemophilia with an inhibitor is never a one-size-fits-all situation. During this session, experts will discuss past, current, and future advancement options for treating hemophilia with inhibitors along with hot research topics.
The VWD Guidelines and Me: Points of Shared Decision-Making
The new VWD guidelines were published in 2020 and heavily integrate the patient voice. But how can you use these in your conversations with healthcare professionals? This session will take a deeper dive into the specific recommendations, where patients and healthcare professionals need to decide together on the best plan of care. You will learn essential skills in shared decision-making to advocate for the care you need.
Current Treatments for Ultra-Rare Bleeding Disorders
This session will discuss current treatment options for rare factor deficiencies and platelet dysfunctions.We Will provide practical tips about effective discussion with your hematology care provider.
Getting to 2030 Blue Sky Vision: A Community Conversation
In the past few years, NBDF has shared its vision for 2030 and started to work alongside the community to get there. Join us for a panel discussion and community conversation about the progress to date and what else NBDF needs to consider as it forges ahead in partnership aiming for the best care possible for the inheritable blood disorders community by 2030.
Rare Factor X Deficiency Survey Shows Disparity in Care
Rare Factor X Deficiency Survey Shows Disparity in Care
Initial data from the survey were presented at the recent Thrombosis and Hemostasis Summit of North America and NHF's Bleeding Disorders Conference last week in Houston.
New Prophylaxis Approval for REBINYN®
New Prophylaxis Approval for REBINYN®
REBINYN® first received FDA approval in May of 2017.
New Genotyping Recommendations Issued by MASAC
New Genotyping Recommendations Issued by MASAC
The new recommendation describes the proven benefits of genotyping people with hemophilia.
DESMOPRESSIN UPDATE: NOTICE TO PATIENTS – INCORRECT EXPIRATION DATE ON CERTAIN LOT
DESMOPRESSIN UPDATE: NOTICE TO PATIENTS – INCORRECT EXPIRATION DATE ON CERTAIN LOT
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.
Novel Pilot Program to Focus on Genetic Diseases and Precision Medicine
Novel Pilot Program to Focus on Genetic Diseases and Precision Medicine
This new pilot program is designed to advance a diagnostic and precision medicine approach in newborns.
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.
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.
Six New MASAC Documents Issued
Six New MASAC Documents Issued
NHF's Medical and Scientific Advisory Council has issued new documents on topics such as prophylaxis, the use of emicizumab in hemophilia patients, and standards of care for bleeding disorders patients at HTCs.
HANDI Launches New Microsite!
HANDI Launches New Microsite!
The site includes a series of frequently asked questions posed by HANDI patrons.
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!
Public Health for Blood Disorders Series Now Available Online!
Public Health for Blood Disorders Series Now Available Online!
This series was designed to inform and engage the community about the ongoing programming and initiatives of CDC-funded groups that serve those impacted by rare blood disorders.
Communicating with Providers
YOU are your most important advocate in the health care system. If you have a child, you are also their most important advocate to make your concerns heard and understood.
How Do YOU Navigate?
Learning through the lived experiences of others is helpful in navigating the landscape of bleeding disorder care. In this workshop, participants will be given an opportunity to learn through investigating scenarios building on the previously delivered material but also through discussions of the issues they are facing in their own everyday life. By the end, participants will receive practical tips and will feel connected and empowered.
Knowing the Players: Being a Savvy Consumer
Having a bleeding disorder comes with great responsibility to get the best care possible. we will address the responsibility that parents, patients, pharmaceutical, and homecare representatives and more
Choose to Take Control
Being a good consumer is more than just access to products and care, it is also about knowing the do’s and don’ts that impact your daily life with a bleeding disorder. It’s about understanding your disease, choosing a care team, clearly communicating your needs and accepting responsibility for making the decisions that ensure your safety and wellbeing. During this interactive presentation, we will discuss tips, tricks and tools available to help you become a better self-advocate and an overall good consumer.
Looking from the Outside in: Musculoskeletal Ultrasound
Ultrasound of the joints and muscles is slowly becoming a staple in many Hemophilia Treatment Centers to detect bleeding episodes and to follow joint health over a long time.
Can You Handle the Tooth? Oral Health Basics
NBDF provides free quality education on issues that matter to the bleeding disorders community. This video asks if you can handle the tooth oral health basics
Inhibitor Treatments: Now and the Future
NBDF provides free quality education on issues that matter to the bleeding disorders community. This video is on inhibitor treatments: now and the future
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
Communicating with Your Providers: Telehealth or In-person
NBD provides free quality education on issues that matter to the bleeding disorders community. This video is communicating with your providers, telehealth or in person
Basics of VWD
NBDF provides free quality education on issues that matter to the bleeding disorders community. This video is on the basics of vwd
Takeda Announces Approval of Prophylactic Indication for VWD Therapy
Takeda Announces Approval of Prophylactic Indication for VWD Therapy
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.
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.
Allied Health Careers in Hematology: Physical Therapy & Genetic Counseling
Allied Health Careers in Hematology: Physical Therapy & Genetic Counseling
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.
Clinical Study and New Website to Focus on von Willebrand Disease and Pregnancy
Clinical Study and New Website to Focus on von Willebrand Disease and Pregnancy
This new study was developed to increase understanding of how best to manage bleeding during delivery and the postpartum period in women with VWD.
New Data Analysis Sheds Light on Bleeding Patterns in Young VWD Patients
New Data Analysis Sheds Light on Bleeding Patterns in Young VWD Patients
The authors of this study focused primarily on birth characteristics, bleeding episodes, and complications.
Spots, Dots, and Clots Clinic: A designated combined hematology/reproductive medicine clinic at OHSU
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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.
New Training Opportunities to Focus on Fellows and Junior Physicians
New Training Opportunities to Focus on Fellows and Junior Physicians
These trainings will be developed for physicians working in federally recognized hemophilia treatment centers in the U.S.
Prophylaxis Indication Restored for BeneFIX®
Prophylaxis Indication Restored for BeneFIX®
BeneFIX® first received FDA approval in the United States in 1997.
New Nursing Guidelines Available on Administration of Bleeding Disorders Therapies
New Nursing Guidelines Available on Administration of Bleeding Disorders Therapies
These guidelines are readily accessible in PDF format on the NHF website.
El Costo de la Reentrada
Después de un año difícil impulsado por la pandemia de COVID19, esperamos una nueva normalidad. Pero, ¿cómo afecta la reincorporación
Pregunte a los expertos
¿Tiene una pregunta candente que hacerle a nuestro panel de expertos? Esta sesión ofrecerá a los participantes la oportunidad de interactuar, participar y hacer preguntas que inviten a la reflexión a un panel de proveedores de atención médica interdisciplinarios en tiempo real. Esta discusión incluirá Miguel Escobar, MD; Kelli Fraga, PT, DPT; Sabrina Farina, LMSW
Inhibitor Education Series: Ask the Experts
This session will offer participants an opportunity to interact, engage, and ask thought-provoking questions to a panel of interdisciplinary healthcare providers in real time.
Rare Series: Current Treatments for Rare Bleeding Disorders
This session will discuss current treatment options for rare factor deficiencies and platelet dysfunctions. It will also provide practical tips about what information will foster an effective discussion with your hematology care provider about your best treatment plan.
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
Women Bleed Too
Women and girls with bleeding disorders often deal with particular issues and taboos related to diagnosis, symptoms, access to care. In this session, an expert who works to improve the quality of life for women and girls with bleeding disorders will discuss essential information on the challenges of getting a diagnosis and accessing care and treatment. In addition, an affected individual will share their experience as a woman with a bleeding disorder.
What Everyone Who Treats with Factor Needs To Know About Inhibitors
If you treat with clotting factor, you may be at risk of developing an inhibitor. This workshop is for people who do not have an inhibitor but want to learn more about risk. We will explain the basics of inhibitors, how they develop, and how and why you should have a conversation with your healthcare provider about screening and monitoring for an inhibitor.
The NEW VWD Guidelines
2020 is the year that VWD guidelines will be announced. This will be an opportunity to learn, explore, and understand the new measures for treatment, diagnosis, and protocols.
Medicinas caseras
Muchas familias y culturas tienen sus propios remedios caseros "especializados" para tratamientos y curas. Esta sesión explorará cuántos de estos remedios caseros
De lo que todos los que tratan con factor sobre los Inhibidores
Si trata con factor de coagulación, puede estar en riesgo de desarrollar un inhibidor. Este taller es para personas que no tienen un inhibidor pero desean aprender más sobre el riesgo.
Basics: VWD
This session will review the signs and symptoms of von Willebrand disease, discuss genetics and inheritance patterns, and define and compare the three types of VWD
OUCH! Tackling Pain Together
NHF’s Medical and Scientific Advisory Council (MASAC) recently approved new treatment guidelines for providers who administer opioids to treat pain in people with bleeding disorders. Come learn about the new guidelines from expert providers and consumers and what they might mean to you.
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
How to Survive 3-5: Developmentally Appropriate Care for PWBD Ages 3-5
In this session, we will evaluate bleed presentation in children with bleeding disorders at 3 to 5 years of age, discuss educational tools and safety guidelines for caregivers, and analyze developmentally appropriate activities for children with bleeding disorders in this age group.
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.
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.
ACA Marketplaces to Reopen
ACA Marketplaces to Reopen
The special enrollment period lasts from February 15th to May 15th, 2021.
Men? I Feel Like a WOMAN... with a Bleeding Disorder
Expanding your PT evaluation skills to include the unique considerations for women with bleeding disorders.
Just Do It... or Don't
Given relatively limited literature specifically on return to physical activity after injury for people with bleeding disorders, many physical therapists may lack competence in developing tailored recovery plans and providing prudent guidance to patients and their families. We will consider and apply recommended guidelines for return to activity and sport, critically appraise existing return to activity evaluation tools and applications, and compare and contrast return-to-work evaluation tools and applications for people with bleeding disorders.
Nipping Pain in the Bud: The Science Behind Medical Marijuana
Explore the science of medical marijuana as a pain relief option. Learn about the evidence, dispension methods &implications for bleeding disorders patients.
Total Joint Replacement: Full Disclosure
Total joint replacements can be performed safely in people with bleeding disorders. In this session, we will address realistic expectations, possible complications, and potential outcomes of total joint replacement surgery in patients with bleeding disorders.
Demystifying the Iliopsoas: The Boogeyman of All Muscle Bleeds
This session will take a deep dive into identification and management of acute to chronic iliopsoas bleeds. Is the psoas to blame for all hip pain?
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.
Pain Management: Tool Box Not a Pill Box
The session will discuss pain related to bleeding disorders in the developmental stages and how infants, children and adolescents with bleeding episodes experience, express and cope with pain. It will review the latest research on management and assessment of pain in children and provide guidance on pain education for families and caregivers. Treatment and intervention strategies for physical therapists will be reviewed and followed by a group discussion.
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"
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"
Basics of Glanzmann’s Thrombasthenia Bernard-Soulier Syndrome
This pre-recorded session is a part of the virtual Rare Bleeding Disorders Conference. It will explain the diagnosis, symptoms and treatment options of both Glanzmann's Thrombasthenia and Bernard-Soulier Syndrome.
Speaker: Jim Munn, BSN, RN
Basics of Factor 5 Deficiency
This pre-recorded session will explain the diagnosis, symptoms and treatment options of Factor 5 Deficiency, Combined Factor 5 and Factor 8 Deficiency and Factor Five Short, also known as east Texas Bleeding Disorder.
Speaker: Sweta Gupta, MD
Basics of Factor 13 Deficiency
This pre-recorded session is a part of the virtual Rare Bleeding Disorders Conference. It will explain the diagnosis, symptoms and treatment options of Factor 13 Deficiency.
Speaker: Suchitra Acharya, MD
Basics of Factor 7 Deficiency
This pre-recorded session is a part of the virtual Rare Bleeding Disorders Conference. It will explain the diagnosis, symptoms and treatment options of Factor 7 Deficiency.
Speaker: Suchitra Acharya, MD
Basics of Factor 11 Deficiency
This pre-recorded session is a part of the virtual Rare Bleeding Disorders Conference. It will explain the diagnosis, symptoms and treatment options of Factor 11 Deficiency.
Speaker: Danielle Nance, MD
Basics of Factor 10 Deficiency
This pre-recorded session is a part of the virtual Rare Bleeding Disorders Conference. It will explain the diagnosis, symptoms, and treatment options of Factor 10 Deficiency.
Basics of Rare Platelet Disorders
This pre-recorded session is a part of the virtual Rare Bleeding Disorders Conference. It will explain the diagnosis, symptoms, and treatment options of rare platelet disorders.
Speaker: Peter Kouides, MD
Marijuana: Breaking it Down, the Good, the Bad, and the Consumption
*ASWB
Moderators:
Kathaleen Schnur, LSW
Social Worker, Hemophilia Center of Western Pennsylvania
Alfredo A. Narvaez, Jr., MSW, LMSW
Social Worker, Louisiana Comprehensive Hemophilia Center
Tulane University Medical Center
Speaker:
Khalid Namoos
Medical Fellow, UCLA David Geffen School of Medicine
By the end of this session, the participant should be better able to:
Ethics: Blurred Boundaries
*ASWB
Moderators:
Kathaleen Schnur, LSW
Social Worker, Hemophilia Center of Western Pennsylvania
Lucy Ramirez, MSW, LCSW
Clinical Social Worker, Rush Hemophilia & Thrombophilia Center
Speaker:
Alana Brunner, LCPC, NCC, CCMHC
Mental Health Therapist
St. Luke’s Psychiatric Wellness
By the end of this session, the participant should be better able to:
Care Coordination for the Aging Bleeding Disorders Population
*ANCC
Moderator:
Tamara Bullock, RN-BC, BSN
Hemophilia Community Nurse, University of Iowa Hospitals and Clinics
Speakers:
Jeff Cornett, MSN, RN
Vice President of Research and Public Policy
Hemophilia of Georgia
Shanna Mattis, MPH
Public Health Associate and Social Worker
Hemophilia of Georgia Center for Bleeding and Clotting at Emory University
Gynecologic Management for Female with Bleeding Disorders
*ANCC
Moderator:
Kelly Tickle, MSN, APN, PPCNP-BC
Pediatric Nurse Practitioner, Children’s Healthcare of Atlanta
Speaker:
Kalinda Woods, MD, FACOG
Assistant Professor, Department of Gynecology and Obstetrics
Emory University School of Medicine
By the end of this session, the participant should be better able to:
Cannabis Applications & Implications
*ANCC
Speaker:
Khalid Namoos
Medical Fellow, UCLA David Geffen School of Medicine
By the end of this session, the participant should be better able to:
- Compare cannabis formulations and discuss the evidence for their potential use
- Discuss the evidence for clinical indications for cannabis use
- Describe the pharmacokinetics and physiological effects of cannabis
Mild Phenotypes: The Struggle is Real
*ANCC
Moderator:
Jennifer Donkin, RN, DNP, CPNP, CFNP
Nurse Practitioner
Children's Hospital of Los Angeles
Speakers:
Sharon L. Littig, RN
Clinical Coordinator
Cardeza Foundation Hemophilia Center at Jefferson University
Penni J. Smith, MPA, BSN, RN-BC
Hemophilia/Hematology RN Coordinator
Utah Center for Bleeding and Clotting Disorders
Primary Children’s Hospital
Disorders of Fibrinogen
*ANCC
Moderator:
Kimberly Hurdstrom, RN, BSN
Hemophilia & Thrombosis Center, University of Colorado School of Medicine
University of Colorado Denver
Speaker:
Marilyn Manco-Johnson, MD
Director, Hemophilia & Thrombosis Center
Professor, Pediatrics Hematology-Oncology
University of Colorado School of Medicine
University of Colorado Denver
By the end of this session, the participant should be better able to:
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:
Challenging Cases
*ANCC
Moderators:
Sharon L. Littig, RN
Clinical Coordinator
Cardeza Foundation Hemophilia Center at Jefferson University
Jessica Pindilli, BSN, RN
University of Wisconsin Hospital & Clinics
UWHC Comprehensive Program for Bleeding Disorders
Speakers:
Maria Tovar-Herrera, RN-BC, BSN
RN Coordinator
Rush Hemophilia and Thrombophilia Center
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.
Comment Period for VWD Clinical Practice Guidelines Open
Comment Period for VWD Clinical Practice Guidelines Open
NHF is seeking comments on draft ASH ISTH NHF WHF clinical practice guidelines on VWD Diagnosis & Management.