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. Our research strategy is a "Full Spectrum Research" platform that includes Basic, Translational, Clinical and Neuro-Habilitation Research.  Below are simple, brief overviews of each aspect of our research strategy in "everyday" language.


Our Strategy: Overview

What is Neuro-Habilitation Research?


 What is Basic Research?

What is Translational Research?

What is Clinical Research?



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