News

Sickle cell disease is linked to an abnormality in the hemoglobin protein, which normally helps carry oxygen throughout the body via red…

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 published in the European Journal of Haematology describes the challenges inherent in managing both bleeding and excessive clot risk…

This month HANDI offers resources that support mental well-being and connect the bleeding disorders community with timely, practical, and readily accessible information.

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…

Pathway to Cures (P2C) announced its collaborative partnership with Afimmune to support the development of Epeleuton as a potential therapeutic for Sickle Cell Disease…

The U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (Pfizer, Inc.), a new hemophilia B gene therapy product. It is designed with…

Primary immune thrombocytopenia is a rare autoimmune disorder that causes a significant reduction in platelets, which are necessary for effective coagulation. A…

A Green Bay resident became the first Wisconsinite to receive a gene therapy treatment for hemophilia A since it was approved by…

New documents issued by NBDF’s Medical and Scientific Advisory Council (MASAC) cover a wide range of topics relevant to bleeding disorder patients…

Pfizer Inc. announced that its gene therapy Beqvez (fidanacogene elaparvovec-dzkt) has been approved by the US FDA for treating adults with moderate…

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…