• Help people with EB

    Your support of DEBRA International or a national EB group enables us to provide the best quality of life for families and individuals affected by EB.

Donation Account: IBAN AT65 6000 0005 1004 5254, BIC BAWAATWW, DEBRA International, Am Heumarkt 27/1, 1030 Vienna

donate now


Amicus Phase 3 Study in EB (ESSENCE) Nearing Completion of Enrolment

Amicus Therapeutics’ ESSENCE Study of the investigational topical cream for wound healing (SD-101) in EB is rapidly nearing completion of enrolment. It is anticipated that patient enrolment will close in the coming weeks.

Anyone interested in participating in the trial should contact one of the approximately 40 international study sites to see if they might have a qualifying wound.

All sites and contact information can be found at https://clinicaltrials.gov/ct2/show/NCT02384460?term=SD-101&rank=3.

All sites provide travel support including transportation, room and board as needed. With a limited number of enrolment slots still available, people are encouraged to make the contacts as soon as possible.

The upcoming closure of enrolment means that Amicus should have top-line results mid-year in 2017. At that time, Amicus will begin to understand the effect of SD-101 on healing wounds of people with EB and will proceed to evaluate the results of the emerging data and determine the next steps in the development of this investigational product.

DEBRA International provides information and news that may be of interest to people with EB, but does not recommend or endorse products, or participation in any particular clinical trial. We strongly recommend that anyone considering a treatment or clinical trial should also speak with their doctor, or medical team, who helps them manage their EB.