The quest for targeted therapy in fragile X syndrome.

Abstract excerpt

Fragile X syndrome (FXS) is the most common, monogenetic cause of intellectual disability and autism-spectrum disorders. Although there is no effective therapy, greater understanding of disturbed neuronal pathways has introduced options for targeted therapy. But whereas many FXS phenotypes were improved in preclinical studies with drugs targeting these pathways in the FXS mouse model, attempts to translate these...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Disease Models, Animal
• Share to Drug Design
• Share to Fragile X Syndrome