NBDF Announces New Treatment Recommendations for Bleeding Disorders
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Rebinyn Supplies Restored in 3,000 IU Vial Size
Hemophilia B Therapy FDA Approved for Expanded Indication
(Video) Celebrating Nurses in Bleeding Disorders Care
New Training Available to Early Career Hematologists
Von Willebrand Emergency Care Manual Now Available in Spanish
FDA Approves Therapy for Rare Disorder Affecting Red Blood Cells
Novo Nordisk Experiencing Shortage of Rebinyn 3,000 IU Vials
Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial
Yale Researchers Explore the Possibilities of von Willebrand Factor
FDA Approves Pair of Cell-Based Gene Therapies for Sickle Cell Disease
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
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
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
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.
Recent News from Pathway to Cures
Recently, Pathway to Cures (P2C) -- NBDF's venture philanthropy fund -- announced exciting updates. Click below to read important press releases.
International Hemophilia Survey Suggests Psychosocial Challenges and Lack of Shared Decision Making
New Study Looks at Hemophilia Survival Rate Disparities
Two New MASAC Documents Focus Substance Use Facilities Access and Treatment Recommendations
FDA Approves First-Ever Nonprescription Oral Contraceptive
Patient-Clinician Partnerships the Theme of ASCO Presidential Address
Rare Disease Fellowship Program Now Accepting Applications!
New MASAC Documents Approved on Physical Therapy, Gene Therapy Readiness, Vaccine Guidance, and Treatment
Updates on Generic Desmopressin Product
Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage
Guilt in Mothers of Children with Hemophilia Focus of New Study
New Medscape Activity to Help Institutions Offer Gene Therapy
National Hemophilia Foundation Names Michael Recht, MD, PhD, MBA, as Chief Medical and Scientific Officer
WFH Gene Therapy Registry Goes Live
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.
New Document to Help Residential Substance Use Disorder Placement for Individuals with Bleeding Disorders
Update for the Hemophilia Community
Virtual Grand Round Series to Focus on Hemophilia Gene Therapy
PPTA Posts Statement on Monkeypox and Plasma Protein Therapies
New Genotyping Recommendations Issued by MASAC
National Hemophilia Foundation Requests Risk Evaluation and Mitigation Strategy for Pending Gene Therapy Treatments
FOR IMMEDIATE RELEASE
July 8, 2022
DESMOPRESSIN UPDATE: NOTICE TO PATIENTS – INCORRECT EXPIRATION DATE ON CERTAIN LOT
Novel Pilot Program to Focus on Genetic Diseases and Precision Medicine
Bone Health in VWD and Hemophilia Carriers the Focus of Retrospective Study
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
NHLBI Grant Supports Scientific Research to Enhance Hemophilia A Gene Therapies
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!
New Medscape Activity Seeks to Prepare Clinicians for Gene Therapy
New Study Looks at Physical Activity Risk in Patients with Hemophilia A
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
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
New Data Analysis Sheds Light on Bleeding Patterns in Young VWD Patients
New Training Opportunities to Focus on Fellows and Junior Physicians
Q&A with My Life, Our Future Researcher: Dr. Ming Lim
My Life, Our Future (MLOF) is a nationwide campaign offering free genetic testing, or genotyping, for people affected by hemophilia.
Descriptive Study Explores HTC Social Worker Roles
While social workers have functioned as essential members of the multidisciplinary care teams at the core of U.S. hemophilia treatment center (HTC) network since its establishment in 1975, a breakdown of their primary roles, both actual and “ideal,” have not been clearly established.
WFH Humanitarian Aid Program Reaches Milestone
MMWR Report Looks at COVID-19’s Impact in Public Health Workers
Prophylaxis Indication Restored for BeneFIX®
Vector Biology and Hemophilia Gene Therapy the Focus of Review
New Nursing Guidelines Available on Administration of Bleeding Disorders Therapies
UCSD Offers Virtual CME-Accredited Trainings on Musculoskeletal Ultrasound
Learn More About Shared Decision Making in Hemophilia
Shared decision making (SDM), the concept by which patients
Study Looks at Neuraxial Anesthesia and Postpartum Complications in Hemophilia
Updated MASAC Documents Address Females Affected by Bleeding Disorders, Pregnancy, VWD
Medscape Activity Designed to Prepare Clinicians for Arrival of Gene Therapy
New Guidelines on the Diagnosis and Management of VWD
Newly Launched Medscape Activity Looks at Gene Therapy and Nursing
New Medscape Activity Includes Latest Gene Therapy Clinical Trial Updates
A Great Opportunity to Learn More About Rare Platelet and Bleeding Disorders!
Small Study Looks at Use of Epidural Analgesia During Labor in VWD Type 1
A research abstract presented at the recent Thrombosis & Haemostasis Summit of North America 2020 Virtual Conference suggested that epidural analgesia may be a safe option for pregnant women with von Willebrand disease (VWD) type 1, provided clotting factors are maintained at certain levels.