Despite an anticipated price tag of $2.5 million, there s no way to predict who will have long-standing benefit from valoctocogene roxaparvovec and who will not.
A once-weekly prophylactic treatment of this investigational agent led to normal or near-normal factor VIII activity levels in most patients with severe hemophilia A.
A new treatment that helps people with hemophilia A maintain higher levels of a crucial blood clotting factor with fewer treatments is a victory for patients, according to a new editorial in The New England Journal of Medicine this week by Dr. Cindy Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University School of Medicine.
Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases.