Hemophilia B Gene Therapy Trial Data Published
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
Hemophilia B Therapy FDA Approved for Expanded Indication
New Hemophilia Fact Sheet Now Available
Novo Nordisk Experiencing Shortage of Rebinyn 3,000 IU Vials
Study Suggests Hemophilia Patients' Preferences Evolving Along with Treatments
Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial
NBDF and HFA Address Concerns on WHO's Recommendation of Cryoprecipitate
FDA Accepts Biologics License Application for Marstacimab
CSL Presents Three Year Treatment Data for Hemophilia B Gene Therapy
Novo Seven Unavailable in 8 mg Vials Due to Supply Constraints Beginning in January 2024
First Patient Dosed with Hemophilia Gene Therapy Candidate ASC618
Sernova Receives Dual FDA Designations for Investigational Hemophilia A Therapy
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.
International Hemophilia Survey Suggests Psychosocial Challenges and Lack of Shared Decision Making
Investigational T-Cell Therapy for Hemophilia A Inhibitors Receives Orphan Drug Designation
New Study Looks at Hemophilia Survival Rate Disparities
Patient Safety in the Age of Gene Therapy a Central Focus of Review Article
New Trial Data Shows ALTUVIIIO™ Prophylaxis Effective in Children
Hemophilia A Gene Therapy Product Receives U.S. FDA Approval
Patient-Clinician Partnerships the Theme of ASCO Presidential Address
First Hemophilia B Patient Dosed with Gene Therapy Product
Gene Therapy as a Novel Approach to Achieving ITI in FVIII Inhibitors Reviewed
Pfizer Highlights Phase 3 Trial Results of Marstacimab
It is Family: A Conversation with Saylor Behrens
Updates on Generic Desmopressin Product
MLOF Genotyping Project Informed Inhibitor Risk
FDA Requests Additional Information on Concizumab from Novo Nordisk
Sanofi Announces Publication of Two Phase 3 Fitusiran Trials
Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage
Guilt in Mothers of Children with Hemophilia Focus of New Study
BioMarin Announces FDA’s Extended Review of Investigational Hemophilia Gene Therapy
Start the Conversation: Dr. Amy Dunn on Gene Therapy
Start the Conversation: Gene Therapy – An Oasis or Mirage?
New Medscape Activity to Help Institutions Offer Gene Therapy
uniQure Announces Published Trial Results for Approved Gene Therapy
FDA Approves ALTUVIIIO™ for Hemophilia A Patients
Kopenitz family commits substantial funding to NHF health equity initiatives
WFH Gene Therapy Registry Goes Live
Sanofi Announces New Efanesoctocog alfa Data Published in the NEJM
New FIX Chromogenic Assay Launched by Precision BioLogic
Biomarin Provides Hemophilia A Clinical Development Program Updates
Pfizer Announces Trial Updates for Investigational Hemophilia Gene Therapy
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
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. Jyoti Mathur!
Meet Former JGP Fellow, Dr. Seema Patel!
Meet JGP Fellow, Dr. Sean Quinn!
Meet JGP Fellow, Dr. Kaushik Das!
Meet Former JGP Fellow, Dr. Xuejie Chen!
Meet JGP Fellow, Dr. Vishal Srivastava!
Meet Former JGP Fellow, Dr. Elizabeth Chappell, MBA!
BioMarin Shares Community Update for Valoctocogene Roxaparvovec
Recruitment Reopened for Phase 3 AFFINE Gene Therapy Trial
Virtual Grand Round Series to Focus on Hemophilia Gene Therapy
FDA Grants Priority Review for Sanofi’s BIVV001
BioMarin Receives Conditional Approval in Europe for Gene Therapy
BioMarin Presents Abstract on Vector DNA Transmission Risk at ISTH
New Prophylaxis Approval for REBINYN®
Paper Features Q&A Resource on Shared Decision Making and Gene Therapy
Sanofi Provides Fitusiran Trial Updates at ISTH
Sanofi and Sobi® Report Progress with BIVV001 at International Congress
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
Diabetes Risk in Hemophilia vs Unaffected Groups Compared in Retrospective Study
Bayer Announces Discontinuation of Kogenate® FS
FDA Grants Breakthrough Status to BIVV001
BioMarin Announces Delay in Application to FDA
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
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
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
First Patient Dosed in Freeline’s Dose-Confirmation Trial for Hemophilia B Gene Therapy
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
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
New Study Looks at Physical Activity Risk in Patients with Hemophilia A
Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks
uniQure and CSL Behring Announce Trial Updates for Hemophilia B Gene Therapy
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
Spark Announces Multiyear Data Update for Investigational Gene Therapy
New Paper Highlights Integrated Care Model and the National HTC Network
FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
Ameritas Awards Grant to the National Hemophilia Foundation
Patient-Reported Data Informs New Study on Sexual Health and Hemophilia
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.
Prophylaxis Indication Restored for BeneFIX®
FDA Places Clinical Hold on Sigilon’s Investigational Hemophilia Therapy
Kevin Child Scholarship Deadline Set for August 2nd!
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
A new paper published in The Journal of Haemophilia Prac
Article Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy
BioMarin Presents Positive Safety and Efficacy Data for their Hemophilia A Gene Therapy
New Inhibitor Study Using MLOF Research Repository Data Published
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
uniQure Announces End of Clinical Hold on Hemophilia B Gene Therapy Program
uniQure Announces Findings from HCC Case Investigation
Basics: Hemophilia
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
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
Spark Announces Preliminary Data from Investigational Gene Therapy Trial
Octapharma Announces Final Results of Nuwiq® Study
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
Recall Notice: Voluntary Recall Mononine Coagulation Factor IX (Human)
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
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
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: