News
FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
by: Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and…
Defining Gene Therapy for Patients
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Gene therapy is a welcome treatment for patients, which has been anticipated for decades. But how do parents and patients discuss this…
What May Be on the Horizon for Hemophilia Treatment?
Industry News & Research
Gene therapy research continues to evolve. As more information becomes available, the below may help you better understand gene therapy and shared…
In Loving Memory of Neil Frick, NBDF’s Senior Vice President for Medical Programs and Information
GLHF News, Industry News & Research
From the National Bleeding Disorders Foundation’s President and CEO, Phil M. Gattone, M.Ed. It is with profound sadness that we share the…
Product Update from Novo
Industry News & Research
Novo Nordisk sent a letter to NBDF sharing more information about the current product issue related to NovoSeven® RT and Novoeight ®….
Free Educational Videos on the use of Novel Non-Factor Therapies
Industry News & Research, Living with a Bleeding Disorder
The National Bleeding Disorders Foundation (NBDF) is pleased to announce the availability of a series of educational videos on the use of…
Learn About Clinical Issues in Women with Bleeding Disorders
Industry News & Research, Women with Bleeding Disorders
The National Bleeding Disorders Foundation (NBDF) is pleased to announce a series of educational videos on clinical issues in women, girls, and…
Study Provides New Data on Neurodevelopment in Younger Hemophilia Population
Industry News & Research
It has been several decades since the publishing of large U.S. studies of neurodevelopment and other cognitive functions in children and young…
Activity Explores Non-Factor Replacement Therapy in Hemophilia Care
Industry News & Research, Living with a Bleeding Disorder
The National Bleeding Disorders Foundation (NBDF) is excited to announce a new accredited educational opportunity for healthcare providers. This live/virtual program will…
New Study Suggests Prophylactic Treatment May Reduce “Brain Bleed” Risk for People with Hemophilia A
Industry News & Research
Results of a new study published in the Journal of Blood Medicine (JBM), indicate that people with hemophilia A (HA) who have…
First Sickle Cell Patient to Receive Gene Therapy Featured in NYT
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Sickle cell disease is linked to an abnormality in the hemoglobin protein, which normally helps carry oxygen throughout the body via red…
FDA Approval Includes Full ALTUVIIIO™ Pediatric Trial Results
Industry News & Research
The U.S. Food and Drug Administration (FDA) has approved updated prescribing information for ALTUVIIIO™ (Sanofi) to include the full results of the phase…
A New Review on Primary Immune Thrombocytopenia
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
A new review published in the European Journal of Haematology describes the challenges inherent in managing both bleeding and excessive clot risk…
May is Mental Health Awareness Month
Industry News & Research, Living with a Bleeding Disorder
This month HANDI offers resources that support mental well-being and connect the bleeding disorders community with timely, practical, and readily accessible information.
NBDF Supports WFH Statement on ISTH Hemophilia Treatment Guidelines
Industry News & Research, Living with a Bleeding Disorder
The NBDF concurs with WFH’s assessment that the proposed guidelines risk setting hemophilia treatment back 30 years. The National Bleeding Disorders Foundation (NBDF) echoes…
P2C Announces Partnership to Support Development for Sickle Cell Disease
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Pathway to Cures (P2C) announced its collaborative partnership with Afimmune to support the development of Epeleuton as a potential therapeutic for Sickle Cell Disease…
FDA Approves Second Hemophilia B Gene Therapy Product
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
The U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (Pfizer, Inc.), a new hemophilia B gene therapy product. It is designed with…
New Review Weighs Bleeding and Clotting Risk in ITP
Health and Well Being, Industry News & Research
Primary immune thrombocytopenia is a rare autoimmune disorder that causes a significant reduction in platelets, which are necessary for effective coagulation. A…
Green Bay Resident Became First Wisconsinite to Receive Gene Therapy Treatment for Hemophilia A
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
A Green Bay resident became the first Wisconsinite to receive a gene therapy treatment for hemophilia A since it was approved by…
NBDF Announces New Treatment Recommendations for Bleeding Disorders
Industry News & Research, Living with a Bleeding Disorder
New documents issued by NBDF’s Medical and Scientific Advisory Council (MASAC) cover a wide range of topics relevant to bleeding disorder patients…
Pfizer Announces its Gene Therapy, Beqvez, Approved by US FDA
Industry News & Research, Living with a Bleeding Disorder
Pfizer Inc. announced that its gene therapy Beqvez (fidanacogene elaparvovec-dzkt) has been approved by the US FDA for treating adults with moderate…
More Gene Therapy Trials from New Players
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
ReciBioPharm and GeneVentiv Therapeutics are developing GENV-HEM, a gene therapy for hemophilia patients, including those with inhibitors. ReciBioPharm will help GeneVentiv take GENV-HEM from preclinical development to Phase 1/2…
Medexus Pharmaceuticals Received FDA Approval for Hemophilia B Treatment, Ixinity®
Industry News & Research, Living with a Bleeding Disorder
Medexus Pharmaceuticals has received FDA approval for their hemophilia B treatment, Ixinity®, for children under 12. This approval, based on positive Phase…
Does Your HTC Administer Gene Therapy?
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Not every HTC administers gene therapy. CSL Behring has updated their website so that patients can locate HTC’s that have its gene…