Looking back at a lifesaving enzyme for rare blood disease

This study looks back at medical records to understand how a manufactured enzyme therapy helped people with a rare, inherited blood disorder called congenital TTP. The therapy, called rADAMTS13, is designed to replace a missing enzyme to help prevent dangerous blood clots. Researchers will review data from about 94 people who received this therapy through a special early access program to see how it affected their health, symptoms, and hospital visits.