News

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 has received FDA approval for their hemophilia B treatment, Ixinity®, for children under 12. This approval, based on positive Phase…

Not every HTC administers gene therapy. CSL Behring has updated their website so that patients can locate HTC’s that have its gene…

HFA reports that the Centers for Medicare and Medicaid Services (CMS) has formally extended the special enrollment period (SEP) created during the…

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…

Did you know? Gene therapy is the use of genetic material to treat or prevent disease. Research into gene therapy for hemophilia…

Although it was eagerly anticipated, commercial sales of gene therapy are disappointing. BioMarin’s hemophilia treatment Roctavian had only one US patient last year….

The National Bleeding Disorders Foundation (NBDF) HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding…

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…

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…

Takeda recently announced positive results from a phase 2 clinical trial of their investigational therapy (Mezagitamab), to treat patients with persistent or…

Hereditary factor X deficiency is an ultra-rare bleeding disorder that occurs in approximately 1 in 500,000 to 1 in a million people…

The fact sheet includes basic information about hemophilia. The National Bleeding Disorder Foundation’s (NBDF) HANDI Resource Center is pleased to announce the…

Learn why experts consider the term to be a misnomer. by: Donna Behen Look up the word “mild” in the dictionary, and…

Each March, the community calls attention to inheritable blood and bleeding disorders during Bleeding Disorders Awareness Month. This time gives patients and…

NBDF is pleased to announce that the Emergency Care for Patients with von Willebrand Disease treatment manual is now available in a Spanish language…

A new study published in the journal Blood Coagulation & Fibrinolysis, looked at the impact of hereditary factor X deficiency (HFXD) on patients…

Infections from respiratory viruses, including the latest COVID-19 variants, are increasing this winter—but vaccines offer effective safeguards for people with inheritable blood…

The U.S. Food and Drug Administration (FDA) has approved Fabhalta® (iptacopan), the first oral therapy for the treatment of adults with a…

Novo Nordisk recently informed NBDF that they are experiencing a temporary shortage of their product Rebinyn®, specifically in the 3,000 IU vial…

In recent years, the arrival of novel hemophilia treatments that are effective and less burdensome to administer have opened up new possibilities…

Takeda today announced that it is conducting a voluntary market withdrawal for two product lots of 650 IU VONVENDI® [von Willebrand factor]…

Roctavian™ was approved in the summer of 2023 for the treatment of adults with severe hemophilia. The first hemophilia A gene therapy…

Takeda announces a voluntary withdrawal for 650 IU VONVENDI® due to misprinted labels showing incorrect expiration dates. Takeda announced today that it…