Rett syndrome (RTT), a unique neurodevelopmental disorder seen almost exclusively in females, was first recognized about 50 years ago by Andreas Rett , a developmental pediatrician in Vienna, and virtually simultaneously by Bengt Hagberg, a Swedish neurologist. However, not until a chance meeting of the two in 1981 were these observations crystallized, yielding the first widely read English-language description of RTT . Thereafter, RTT received considerable attention throughout the world. Concerted effort led to the identification of mutations in the gene Methyl-CpG-binding protein 2 (MECP2) in 1999 , whereupon laboratory-oriented research exploded, including the development of numerous animal models. A key observation from an early mouse model indicated the possibility of substantial reversibility of the disease phenotype . Since then, translational research has been conducted in available mouse models to identify potential therapies including insulin-like growth factor 1 (IGF-1) , disabling GABAergic neuron function , and stem cell transplantation . Recently, intriguing results showed that alterations in cholesterol metabolism in response to statin therapy had a beneficial effect on RTT-associated symptoms in a mouse model of the disease. This has prompted interest in advancing this treatment to humans.