Our Research Strategy

Our objective is to accelerate outstanding discoveries in fundamental Rett syndrome research toward clinical testing with the goal of improving the lives and best practices to treat those diagnosed with Rett syndrome.  Our emphasis on translational and clinical research will ensure that new ideas are constantly flowing into the drug pipeline and neuro-habilitation platforms aiming at accelerating the treatments for Rett syndrome.

Watch: Dr. Steven Kaminsky, CSO, outlines our Research Strategy.


Our Model

We are building a better understanding of the therapeutic needs for Rett syndrome through the Rett Natural History Study and implementing a translational research agenda based on the key biology that needs to be targeted for treatment.  Our efforts are in two areas:

  • Research exploring specific pharmacologic approaches that could contribute to the development of drugs correcting the biology of Rett syndrome.
  • Research to develop tools and resources that will help reset the neurological development in Rett syndrome.

Through our annual call for proposals we fund new ideas identifying relevant biological targets and promising therapeutics that can benefit the Rett syndrome community.  Our current clinical research and drug trials are the largest efforts worldwide to change the clinical course of Rett syndrome.

Programs to Fulfill Our Research Plan

The Basic Research Program 
To correct the biology we first must understand what has gone wrong

The Translational Research Program   
To develop treatments that change the biology of Rett Syndrome

The Scout Program
To accelerate the testing of compounds and move ideas into medicines

Read more about The Scout Program

The Neuro-Habilitation Program 
To reset the neurology we must find methods to habilitate cognition, speech, occupational and physical activities

The Natural History Study and Clinical Trials Network Program   
To build a national clinical trials network through a partnership with the federal government and fully understand the clinical aspects of Retts syndrome while conducting clinical trials aimed to correct Rett biology

The Mentored Training Fellowship Program   
To build the lab bench of future Rett syndrome researchers