The Fondation Pictet financially supports EspeRare’s new programme in a disabling and rare skin disease called X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED). EspeRare is honoured that such a prestigious foundation acknowledges this new project, designed to cure the most threatening symptoms of this disease.
XLHED is a serious rare disease that is life-threatening, particularly in the first years of life. Infants affected by this genetic disorder cannot sweat and are at risk of sudden death due to hyperthermia and severe respiratory issues. See here for more information on the disease.
This new programme, called EspoiR-004 (ER-004) is the first and only therapy for this disease. It is also one of the first treatments designed to be delivered in-utero, paving the way for other such treatments.
