The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.
The treatment is not cheap: Roctavian will cost $2.9 million for a single infusion, the
An experimental injectable drug appears effective in reducing bleeds in patients with hemophilia A and B, according to a pair of new clinical trials.
Two-thirds of people with treatment-resistant hemophilia who were treated with the drug fitusiran had no bleeds at all af...
People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.
The U.S. Food and Drug Administration approved the new gene therapy Hemgenix on Nov. 22. Soon after, drugmaker CSL Behring revealed its cost.
People with hemophilia B could find their bleeding risk dramatically reduced with just one injection of an experimental gene therapy, a new study reports.
Hemophilia B is a rare and inherited ...
Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report.
Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It's caused by a mis...
Researchers may have found a way for people with severe hemophilia to take their standard treatment less often, if the results of an early trial pan out.
In what experts called a feat of bioengineering, scientists were able to create a "fusion protein" that may exten...
A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.
Patients who received the one-time intravenous therapy continue to have a more than 90% decrease in bleeding events two to three ...