Cell Phones and Landlines: The Impact of Gene Therapy on the Cost and Availability of Treatment for Hemophilia

Abstract

Hemophilia treatment evolved rapidly in the latter half of the twentieth century, a period during which the disease went from one that was nearly always fatal in childhood or adolescence for those severely affected to one that is now, although still a chronic condition requiring lifelong medical management, characterized by a nearly normal life span and quality of life for those who have access to optimal treatment.1