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…
FOR IMMEDIATE RELEASEMEDIA CONTACTAngelina Wangawang@hemophilia.org(212) 328-3767December 6, 2023NBDF Announces 2023 Kevin Child Scholarship WinnerNEW YORK - The National Bleeding Disorders…
Octapharma’s human plasma-derived von Willebrand Factor (VWF)/Factor VIII replacement product Wilate® has been granted expanded approval for prophylaxis by the U.S. Food and Drug Administration (FDA) to include all types of von Willebrand disease (…
Federal
Copay Accumulator Adjusters: On Nov. 15, the US Department of Health and Human Services (HHS) issued the proposed 2025 Notice of Benefit and Payment Parameters. Despite the advocacy of…
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At Novo Nordisk, we understand that our medicines are critically important to patients. As part of our commitment to sharing timely and transparent information, we want to provide you with an important update about the NovoSeven® RT (coagulation…
Paxton Mills is one of the newest members of NYLI, of the 2023 cohort. Paxton has been actively involved in the inheritable blood and bleeding disorders community since her diagnosis with having a rare platelet disorder. Watch the video or read the…