EspeRare participates to the National Foundations For Ectodermal Dysplasias (NFED) 2019 family conference
At EspeRare, we are committed to improve the lives of children with life threatening rare diseases. EspeRare addresses the unmet medical needs of these children by uncovering the potential of existing treatments. As a not-for-profit organization, we achieve this through a collaborative approach centred on patient engagement with the aim of giving children and their families fair access to these therapies and a new hope for the future.
In line with EspeRare mission to work closely with the patients community, we will present at this patients family conference, the latest update on our most advance programme and our plan to conduct an in utero clinical development for the treatment of a rare form of ectodermal dysplasia (XLHED). This treatment has not only the capacity to correct the most debilitating symptoms of XLHED, but also setting a novel regulatory path for treating genetic diseases before birth.