What is
Edelife?
As you know, there is currently no treatment for Ectodermal Dysplasia (XLHED).
Pierre Fabre and the EspeRare Foundation, known as the Sponsor of the study, are conducting a research study or clinical study, called EDELIFE, on the safety and possible health benefits of an experimental medicine for XLHED called ER004.
Earlier studies
Previously, six XLHED-affected boys received the ER004 treatment before birth (pre-natal treatment). After birth, all six babies showed improvement in some symptoms, particularly regarding their ability to sweat.
Some of these results have been reported in a scientific journal (N Engl J Med 2018; 378:1604-1610↗).
Some of these results have been reported in a scientific journal (N Engl J Med 2018; 378:1604-1610↗).
These prenatal treatments using ER004 indicated the potential of this experimental medicine to prevent or at least significantly reduce the severity of some XLHED symptoms.
Objectives of the EDELIFE study
We now want to confirm these preliminary results in a robust clinical study, with 2 aims:
- We want to find out if prenatal treatment with ER004 is well tolerated
- We want to find out if prenatal treatment with ER004 can lead to a long-lasting improvement of some symptoms in XLHED-affected boys
If successful, this study could lead to ER004 being approved as the first commercially available treatment for XLHED.