News

Orphan drug designation is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States. The…

Some compelling results from the ‘Hemophilia Life Stages and Changes Global Survey’ were recently announced in a new press release. The survey…

Marstacimab (PF-06741086) is under development for the treatment of hemophilia A and hemophilia B. It’s administered through intravenous or subcutaneous route. It…

Learn about bypassing agents as treatment for inhibitors and whether they might be right for you. One of the most problematic issues…

Orphan drug designation is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States. The…

From Curiosity To Eligibility And Beyond! Gene therapy has arrived! Join us for a one-hour presentation to hear how gene therapy is…

Analysis focused on 90 men with hemophilia A and B in Turkey by Lindsey Shapiro, PhD | September 15, 2023 About a quarter (24.4%) of…

Type 1 plasminogen deficiency, also known as hypoplasminogenemia, is a very rare genetic disorder. A recent publication in the journal Haemophilia, represents a…

There are several programs and resources designed to assist individuals and families grappling with the financial burden of living with an inheritable…

A new commentary, “Closing the Diagnostic Gap in Adolescents and Young Adult Women with Bleeding Disorders,” published in the August issue of…

Yesterday, the U.S. Food and Drug Administration approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A. This new treatment option, for the treatment of…

Takeda, in agreement with the U.S. Food and Drug Administration (FDA), has decided to voluntarily replace BAXJECT® II reconstitution devices produced by…

MASAC issued to two new documents during the NBDF’s Bleeding Disorders Conference this past August. The National Bleeding Disorders Foundation (NBDF) is…

Learn more about these treatments for inhibitors. One of the most problematic issues that can occur with hemophilia are inhibitors—a complication that happens…

Is donating plasma safe when you have a severe bleeding disorder? In most cases, the answer is yes! Plasma fully regenerates in…

In 1948, the foundation got its start as simply “The Hemophilia Foundation” – then in 1956, the foundation formally incorporated into what…

Roche is discontinuing its investigational hemophilia A gene therapy called SPK-8016. (SPK refers to Spark Therapeutics, which is carrying out trials) But clinical…

Alhemo™ (concizumab injection) is used for hemophilia B patients who have developed inhibitors, which prevent replacement factor IX therapies from working properly….

As of 2022, the federal “No Surprises Act” protects people covered under group and individual health plans from receiving surprise medical bills…

The world’s first book on the world’s most commonly inherited bleeding disorder, by Laureen A. Kelley and Paul Clement, is back in…

Advocacy groups representing various disease communities voice concerns over Payer Matrix’s classification as a ‘Patient Advocacy Company.’ Read about their viewpoints and…

Takeda recently raised an issue with some BAXJECT® II reconstitution devices produced between October 2021 and January 2022 for use in conjunction…

In an effort to ensure that all copays count with insurance companies, and address the challenges of Copay Accumulator Adjustors, the Wisconsin…

“Insurance companies assert that restricting accumulator and maximizer programs will cause higher health insurance premiums for everyone, but that claim is false….