A prenatal study for XLHED affected boys
A prenatal study for XLHED affected boys
A prospective, open-label, genotype-match controlled, multicenter clinical trial to investigate the efficacy and safety of intra-amniotic ER004 as a prenatal treatment for male subjects with X-linked hypohidrotic ectodermal dysplasia (XLHED)
Brief summary
This is a prenatal trial for X-linked Hypohidrotic Ectodermal Dysplasia (XLHED) affected boys. The purpose of the trial is to evaluate how prenatal treatment of male fetuses with ER004 works and how it is tolerated. ER004 is an experimental medicine for XLHED not yet approved for use in humans, except in clinical studies.
ER004 is made in a laboratory to replace an important protein called EDA1 (Ectodysplasin-A1), missing in people affected by XLHED. Absence of this protein during the baby’s development in the womb leads to some important structures not forming properly (sweat glands, hair, teeth etc).
EspeRare Foundation
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