For centuries, hemophilia was a dangerous genetic disorder with no treatment. Children with hemophilia often didn't make it to adulthood, since a bump or fall could cause massive internal bleeding.
Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...
Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?
Hemophilia is a hereditary blood disorder characterized by a deficiency of certain coagulation factors, which can lead to life-threatening bleeding if left untreated. Why hemophilia A (with factor ...
Sangamo Therapeutics regained development and commercialization rights for its hemophilia A candidate, giroctocogene fitelparvovec, after partner Pfizer conveyed its decision to terminate their ...
Saliva activates coagulation in persons with hemophilia A. ScienceDaily. Retrieved February 1, 2025 from www.sciencedaily.com / releases / 2025 / 01 / 250114125253.htm Medical University of Vienna.