X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED), the most common form of ectodermal dysplasia, is caused by a broad spectrum of mutations in the ectodysplasin A1 (EDA-A1) gene. The main symp- toms of XLHED are hypo- or anhidrosis, oligo- or anodontia and hypotrichosis. Current treatment options are limited to managing disease symptoms and complications. Effective corrective treatment for XLHED remains a high unmet medical need.
Edelife is a clinical study of the safety and possible health benefits of an investigational medicine for pregnant women expecting an ectodermal dysplasia (XLHED) affected boy.
The Edelife investigational treatment, ER004, consists of a signaling protein replacement molecule designed for specific, high affinity binding to the endogenous receptor, intended to replace missing EDA-A1 protein in utero for patients with XLHED.
Administration at several specific points in pregnancy is essential, as ER004 impacts development of ectodermal precursors of sweat glands, teeth, etc.
Compassionate Use of ER004 in XLHED Patients
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This is a website about the Edelife Clinical Trial dedicated to Healthcare Professionals.