What Research is Being Done to Find a Cure or Additional Treatments?

Within the Federal Government, two institutes – the National Institute of Neurological Disorders and Stroke (NINDS) and the National Institute of Child Health and Human Development (NICHD), support clinical and basic research on Rett syndrome.

Understanding the cause of this disorder is needed to develop new therapies to manage specific symptoms, as well as for providing better methods of diagnosis. An animal model for Rett syndrome has been developed in mice and several therapeutic strategies have resulted in improvements in the symptoms these mice experience including breathing control and cognition. While these findings provide hope, they do not necessarily represent a cure in these mice nor is their translation to treatment of individuals with Rett syndrome necessarily going to result in similar impacts given many factors including the complexity of brain development and the fact that these strategies would likely be implemented at diagnosis which is late in terms of brain development. Additionally there are significant technical issues to be overcome in trying to implement these treatments in humans. At the time of this writing (2013) two agents were moving into clinical trials for treatment of Rett Syndrome NNZ-2566 and IGF-1. These trials will test the safety of these agents and whether these compounds can reverse some of the symptoms or modify the progression of Rett syndrome. More information can be found at WWW.Rettsyndrome.org.  Parents can also go to ClinicalTrials.gov to find out if any NIH sponsored trials are available or ongoing. 

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International Rett Syndrome Foundation