The U.S. Food and Drug Administration (FDA) has approved a recombinant enzyme replacement therapy for people with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood disorder. Adzynma, which is manufactured by Takeda…
Get a glimpse into the world of healthcare providers caring for ultra-rare bleeding disorders patients. Our panelists, with diverse backgrounds, will share their experiences, highlight unique challenges, and emphasize the importance of patient…
Explore the main findings from the Women, Girls, and People who have or had the Potential to Menstruate (WGPPM) Summit 2023, including actionable recommendations emerging from the WGPPM Summit white paper. Hear from an engaging panel of Summit…
Come discuss some of the leading advancements in care for the bleeding disorders community.
NBDF's Wednesday Webinars are a free education series open to providers and community members. Register to attend and learn about the latest in research,…
November is recognized as Transgender Health Awareness Month. This is a time to reflect on the experiences of transgender and gender nonconforming individuals seeking and receiving health care. It is also an important reminder of…
Join us Thursday, Dec. 7, at 2 p.m. ET for the next Research Journal Club with Tammuella Chrisentery-Singleton, MD, the chief science officer at the American Thrombosis and Hemostasis Network. She will discuss a recent study published in…
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for an investigational hemophilia A therapy being developed by Sernova, a Canadian-based clinical-stage biotechnology…
November is recognized as Transgender Health Awareness Month. This is a time to reflect on the experiences of transgender and gender nonconforming individuals seeking and receiving health care. It is also an important…
The first patient has been dosed in ASC Therapeutics clinical trial of ASC618, the company’s investigational AAV8 vector-based gene therapy for patients with severe and moderately severe hemophilia A.
Investigators of the phase I/II trial…