Data from clinical trials suggest that the vast majority of the patients respond to the gene therapy for hemophilia A, says Courtney Thornburg, M.D., M.S. But there is also decreasing factor expression.
Second of four parts
In this segment of a video interview with Managed Healthcare Executive, Courtney Thornburg, M.D., M.S., discusses the durability of Roctavian (valoctocogene roxaparvovec) in producing factor VIII and liver inflammation that is treated with corticosteroids.
Thornburg, the medical director of the Hemophilia and Thrombosis Treatment Center at Rady Children’s Hospital-San Diego, was the lead author of a review article about hemophilia gene therapy that was published in July in the journal BioDrugs.