Investigational Hemophilia A Gene Therapy Shows Sustained Benefit

An experimental gene therapy for hemophilia A remained effective up to 3 years after a single infusion, researchers recently reported in the New England Journal of Medicine. BioMarin Pharmaceutical ’s valoctocogene roxaparvovec encodes factor VIII, the blood-clotting protein that’s missing or low in people with hemophilia A, the most common form of the disease.
Source: JAMA - Category: General Medicine Source Type: research