News

Slowdown of Gene Therapy Research Spurs Call to Action
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Global organizations unite to urge collaborative approaches to reducing barriers to advancement of care. The National Bleeding Disorders Foundation (NBDF) has joined…

Two Products to be Discontinued
Industry News & Research, Living with a Bleeding Disorder
Takeda announced that it will globally discontinue two of its hemophilia A treatments: Hemofil M, a plasma-derived therapy, and Recombinate, the first…

Important New Product Launched
Industry News & Research, Living with a Bleeding Disorder
The U.S. FDA has approved Sanofi’s Qfitlia (fitusiran), the first antithrombin-lowering therapy for routine prophylaxis to prevent or reduce the frequency of…

HHS Terminates Division of Blood Disorders and Public Health Genomics
Advocacy & Legislation, GLHF News, Industry News & Research, Living with a Bleeding Disorder
We need your help! Contact your representatives today! On April 1, the US Department of Health and Human Services (HHS) issued termination…

Register for the Wisconsin Bleeding Disorders Conference by May 1!
Advocacy & Legislation, GLHF News, Industry News & Research, Living with a Bleeding Disorder
Whether you’re new to the bleeding disorders community or have been a part of it for years, GLHF’s Wisconsin Bleeding Disorders Conference…

NBDF Enhances Health Access with Strategic Department Expansion
Advocacy & Legislation, Industry News & Research, Living with a Bleeding Disorder
NBDF Expands Health Access Initiatives Through Strategic Department Evolution Foundation Continues Mission Alignment with Enhanced Focus on Health Access, and Innovation to Better Serve Growing…

CDC Changes Affect Bleeding Disorders Community
Advocacy & Legislation, Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
A Message from Phil Gattone, NBDF President and CEO Major restructuring at HHS has placed nearly all CDC Blood Disorders Division staff…

New Paper Looks at Bone Health Assessment in Bleeding Disorders
Industry News & Research
A newly published journal article examined current practices of HTCs for screening and assessing bone health in people with hemophilia and von…

FDA Approves Qfitlia™ – New Treatment Option for Hemophilia A & B
Industry News & Research, Living with a Bleeding Disorder
The FDA has approved Sanofi’s Qfitlia™ (fitusiran), a new subcutaneous therapy for people with hemophilia A or B, with or without inhibitors….

The Bleeding Disorders Community Needs Your Help to Prevent Cuts to Federal Health Programs
Advocacy & Legislation, GLHF News, Industry News & Research
Earlier this week, the Trump Administration announced drastic changes and reductions to the federal health programs that support the bleeding disorders community….

A Gene Therapy Discontinued
Advocacy & Legislation, Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Less than a year after FDA approval, Beqvez, a gene therapy for hemophilia B, has been discontinued. Manufactured by Pfizer, the one-time…

NBDF’s New Video Series: Voices for Policy Change
Advocacy & Legislation, Industry News & Research, Living with a Bleeding Disorder
“Voices for Policy Change” is an on-line resource designed to educate, empower and mobilize on important legislative issues related to bleeding disorders…

Leadership: Anger, Respect, and Results
Industry News & Research, Living with a Bleeding Disorder
by: Laurie Kelley March is hemophilia awareness month and the recent upheaval over leadership in this country has me thinking about leadership…

Pfizer Halts Hemophilia B Gene Therapy
Industry News & Research
In a surprising development, Pfizer has discontinued Beqvez™, their hemophilia B gene therapy, despite FDA approval just last spring. The company cited…

First von Willebrand Disease Patient Dosed with Investigational Sub-Q Therapy
Industry News & Research
Hemab Therapeutics has begun dosing in their phase 1/2 clinical trial of HMB-002, a subcutaneous prophylactic therapy designed for all types of…

Takeda Announces Discontinuation of Two of its Hemophilia Treatments: HEMOFIL® M and RECOMBINATE®
Industry News & Research
On March 18, 2025, Takeda Pharmaceuticals announced it will globally discontinue two of its hemophilia treatments: HEMOFIL® M [Antihemophilic Factor (Human), Method…

Rest in Peace, First Lady
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
The hemophilia community mourns the passing of Suzanne Massie, age 94, arguably the “first lady” of hemophilia. She and her former husband…

All Your Choices in One Place – Factor Chart Outlines Choices
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Download LA Kelley Communications’ updated Factor Chart to see how many choices you may have, what might be right for you and…

A Message from the Heart of NBDF
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Our Commitment to Health Equity & Inclusive Care At NBDF, our mission is clear—every person and family affected by bleeding disorders deserves…

Clinical Practice and Hemophilia Gene Therapy the Focus of New Accredited Activity
Industry News & Research, Living with a Bleeding Disorder
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational von Willebrand disease (VWD) therapy VGA039. The…

FDA Approves New Non-Opioid Treatment for Pain
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder, Pain Management
The U.S. Food and Drug Administration (FDA) recently approved Journavx™ (suzetrigine) 50 milligram oral tablets, a first-in-class non-opioid analgesic developed to treat…

Watch the 2024 Awards of Excellence Ceremony at the National Bleeding Disorders Foundation Annual Conference
Health and Well Being, Industry News & Research
This inspiring event celebrates the exceptional individuals and organizations making a lasting impact in the bleeding disorders community. Trailblazing healthcare professionals and dedicated advocates were…

Ever Wonder Why Hemophilia B Was Once Called Christmas Disease?
Industry News & Research, Living with a Bleeding Disorder
Did You Know? The name for a well-known bleeding disorder has a surprising origin tied to a 5-year-old boy from Canada. Learn why…

Sanofi Announces Positive Data for Its Investigational ITP Therapy
Industry News & Research
ITP is a rare autoimmune disorder that causes a significant reduction in platelets, which are necessary for clotting. Sanofi has announced positive…