Shared decision-making (SDM) is a process by which clinicians and patients work together to make treatment decisions based on clinical evidence that balances risks and expected outcomes with patient preferences and values. SDM is a process where you…
This past week saw the approval of two new therapies, representing significant scientific breakthroughs in the field of cell-based gene therapy to treat patients with sickle cell disease (SCD), a rare condition historically associated with few…
CSL Behring recently announced three-year post treatment data from clinical trial participants who received a one-time infusion of Hemgenix® (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based hemophilia gene therapy. Hemgenix…
The National Blood Disorders Foundation (NBDF) and the American Thrombosis & Hemostasis Network (ATHN) are collaborative partners that share many key aspects of their missions, most importantly, improving the lives of people affected by…
Pfizer Inc. recently announced that the U.S. Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) for marstacimab, its investigational hemophilia therapy that targets an anticoagulant protein known as tissue factor…
Researchers at the Yale School of Medicine have been studying the key clotting protein von Willebrand factor (VWF), to shed light on the processes by which it is stored and released in response to an injury. As VWF is the deficient protein in many…