Periodic Reporting for period 1 - RTTOPHAGY ("Investigating autophagy enhancement as a therapeutic approach for the treatment of Rett syndrome.")

Mutations in the X-linked MECP2 gene cause Rett syndrome (RTT), a devastating neurological disorder which represents a first cause of severe intellectual disability in 1 out 10.000 girls worldwide. Affected females have an apparently normal development until 6-18 months of...