Connect in Pharma, the organisers of an inauguring event for the European pharma and biotech community pledges to raise 20k Swiss Francs to be donated to EspeRare Foundation for the development of new therapeutic solutions in rare diseases. The event that is designed to address the most critical issues and challenges of the industry in a pivotal time for the packaging market is taking place at Palexpo, Geneva, CH on 14-15 September 2022.
EspeRare Foundation is honored to partner with Ceridwen Hughes, photographer and director of SbD Creative who will launch for the first time her new exhibition, “Days of Rare”, during the event at Geneva. Through the lens of Ceridwen each visitor can experience a glimpse of the challenging and inspiring environment of rare disease.
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.
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.
