Composition for use in the treatment of Angelman syndrome and/or autism spectrum disorder, the use of such composition and a method for manufacturing a medicament for the treatment of Angelman syndrome and/or autism spectrum disorder

摘要

The invention is related to a composition for use in the treatment of Angelman syndrome and/or autism spectrum disorder, wherein the composition comprises a substance that restores deregulated steroid hormone signaling and /or restores the expression levels or neuronal activity of E3 ubiquitin-protein ligase E6APmodulates the steroid hormone signaling in order to compensate for deregulation. Furthermore, the invention is directed to the use of such composition and a method for manufacturing a medicament intended for therapeutic application in the treatment of Angelman syndrome and/or autism spectrum disorder.