Advancing treatments for rare diseases together

The EDELIFE study is open and recruiting participants

05Jul

The first fetal patient recruited in the EDELIFE clinical trial has received three injections of the ER-004 treatment as planned. This experimental treatment (not approved for commercial use yet) is administered through the mother’s abdomen into the amniotic fluid around the baby.

ER-004 has been developed to replace the function of ectodysplasin-A1 (EDA1), a protein essential for the normal development of ectodermal structures in the fetus and missing in patients with XLHED. During this pivotal trial, approximately 15 to 20 unborn baby boys will be treated across 8 investigational centers in Europe and the US to assess the safety and efficacy of the experimental medicine.

A Prenatal Study for XLHED Affected Boys

The EspeRare Foundation and the Pierre Fabre group are co-sponsoring the clinical trial and if successful, ER-004 would be the first prenatal treatment for XLHED-affected boys.

To view the press release please follow the link