News
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…
Extended Time for Coverage from CMS
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
HFA reports that the Centers for Medicare and Medicaid Services (CMS) has formally extended the special enrollment period (SEP) created during the…
Privacy Please!
Advocacy & Legislation, Living with a Bleeding Disorder
Patient privacy continues to be a huge concern worldwide, especially in the healthcare field, and especially online. The states of Washington, Nevada,…
Hemophilia and Poetry
Living with a Bleeding Disorder
To finish up National Poetry Month, let’s consider two more poets with hemophilia. by Richard J. Atwood To me, poetry specializes in the…
NBDF Releases Hemophilia Fast Facts
Industry News & Research, Living with a Bleeding Disorder
NBDF’s announces the availability of a Hemophilia Fact Sheet. This two-page PDF document was created to fill the need for a current, accessible…
Hiking with Hemophilia
Health and Well Being, Living with a Bleeding Disorder
Hiking offers people with hemophilia exercise, fun and exhilaration. People with hemophilia benefit from equipment that can lessen the risk of bleeding…
Hemophilia A vs. B: What’s the Difference?
Living with a Bleeding Disorder
Much is the same when it comes to hemophilia A and B, but a few key differences change how common—and how treatable—they…
Answers to Common Questions Asked about Gene Therapy for Hemophilia
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Did you know? Gene therapy is the use of genetic material to treat or prevent disease. Research into gene therapy for hemophilia…
Countries Across the Globe Join the Gene Therapy Race
Gene Therapy, Health and Well Being, Living with a Bleeding Disorder
Canada Considers Funding Patient Gene Therapy; India Conducts First Human Clinical Trials for Hemophilia A An excellent article explains how Canada’s government…
Gene Therapy Struggles in Market
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Although it was eagerly anticipated, commercial sales of gene therapy are disappointing. BioMarin’s hemophilia treatment Roctavian had only one US patient last year….
New Device Can Diagnose Hemophilia Fast
Health and Well Being, Living with a Bleeding Disorder
A trio of researchers at Chung Yuan Christian University, Taiwan developed a device that can rapidly determine both blood type and whether…
Fear of Needles?
Health and Well Being, Living with a Bleeding Disorder
A phobia to needlesticks is a real thing. Read a great article here about how one father helped his son with hemophilia…
Learn More About 2024 Bleeding Disorders Scholarships
Industry News & Research, Living with a Bleeding Disorder
The National Bleeding Disorders Foundation (NBDF) HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding…
Hemophilia B Gene Therapy Trial Data Published
Industry News & Research
A new paper published in The Lancet Haematology journal includes data from the HOPE-B clinical trial program that is investigating the hemophilia B gene…
Rebinyn Supplies Restored in 3,000 IU Vial Size
GLHF News
NBDF recently learned that Novo Nordisk now has 3,000 IU vial size of Rebinyn® back in stock. Rebinyn is the company’s recombinant…
Hemophilia B Therapy FDA Approved for Expanded Indication
Industry News & Research
The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for IXINITY® (Medexus), a recombinant factor replacement therapy to treat…
Green Bay’s Bets Bloody is May 5! Get Your Tickets Today!
GLHF News
Join us at the Resch Expo on Sunday, May 5 for our 4th annual Green Bay’s Best Bloody – where Green Bay-area bars and…
Video Celebrates Nurses in Bleeding Disorders Care
GLHF News
The National Bleeding Disorders Foundation (NBDF) marked its 75th Anniversary in 2023 by recognizing the contribution of various community stakeholders. As part…
Takeda Announces Interim Trial Results for Investigational ITP Therapy
Health and Well Being, Industry News & Research
Takeda recently announced positive results from a phase 2 clinical trial of their investigational therapy (Mezagitamab), to treat patients with persistent or…
The Joy of Lobbying the Senate on Behalf of my Sons with Hemophilia
Advocacy & Legislation, Living with a Bleeding Disorder
How I advocated for the bleeding disorders community on Capitol Hill. by: Joe MacDonald Last Thursday, I visited Washington, D.C., to join my…
Researchers Look at Impact of Factor X Deficiency on Patients/Caregivers
Industry News & Research
Hereditary factor X deficiency is an ultra-rare bleeding disorder that occurs in approximately 1 in 500,000 to 1 in a million people…