No new therapy without a clinical trial
At present, no effective therapies exist for any form of EB. Although the current number of EB clinical trials being undertaken is still small, the diversity of therapeutic approaches in preclinical development and entering clinical trials is growing rapidly.
In 2006, there was one pilot clinical trial for a gene therapy in one patient – the first time there was proof that the underlying genetic fault in one type of EB could be corrected. Today, there are tens of clinical trials underway or planned, for all types of EB, using many different approaches to therapy, including cell-, gene-, protein- and drug therapies.
Clinical trials registers
There are several official clinical trial websites providing information on EB clinical trials worldwide. These include information about location, clinical lead, and trial sponsor, as well as the trial purpose, how the outcomes will be measured, and inclusion/exclusion criteria.
Links to registers
DEBRA International is the umbrella organisation for more than 50 national DEBRA groups. Find out more
EB-CLINET is a network of EB centres and experts which aims to improve medical care for people with EB and to accelerate the way to a cure by connecting health care professionals working with EB for the purpose of sharing of knowledge and experience. Find out more