Intra-amniotic ER004 as a prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED)

• Unité de recherche: INNOSUISSE
• Numéro de projet: 53573.1 IP-LS
• Titre du projet anglais: Intra-amniotic ER004 as a prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED)

Description succincte (Allemand)

Intra-amniotic ER004 as a prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED)

Description succincte (Anglais)

Intra-amniotic ER004 as a prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED)

Résumé des résultats (Abstract) (Allemand)

ER004 is the first therapy specifically addressing X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder caused by the lack of a functional ectodysplasin A1 (EDA1) protein. Intra-amniotic treatment with ER004 has the potential to switch off symptoms throughout patients# lives.