Programmatic Overview

Research Focus / Knowledge Gaps

We are dedicated to helping develop better treatments to improve the quality of life of individuals with Rett syndrome and ultimately find a “cure.”  Treatment is defined as anything that improves the outcome of conditions caused by Rett Syndrome. In addition to therapeutics this may include assistive technologies, communication, physical therapy and education.

Scout Program

The International Rett Syndrome Foundation has decided to pave a new path for Rett syndrome research.  Our mission is to move discoveries to the clinic.  We believe that the Scout Program will aggressively accelerate the testing of compounds, whether new or repurposed, through standardized preclinical studies in the Rett mouse model.  By using a contract research organization, we will ensure the studies will yield robust and reproducible results.  It will help identify compounds that have potential versus those that will fail.  We want those that fail to “fail fast” so we can put more effort into those with potential. 

Drug Discovery Pipeline

Translational research is the name given to the continuous process of moving the knowledge gained from basic early stage “bench” research through various stages of the drug discovery process. Early stage basic research leads to groundbreaking discoveries that set the stage for development of therapeutics to treat disease.

SMART Initiative

Researchers who study Rett syndrome are increasingly identifying new underlying biological pathways that contribute to the cause of and to the symptoms of the disease. The researchers believe that a chemical or biological compound that influences such a biological pathway (or disease mechanism) that they are studying may have an important effect on Rett syndrome.

Advisory Boards

In May 2009 IRSF announced the appointment of two advisory boards that will serve to help set strategic goals for the advancement of research towards the development of therapeutics for Rett syndrome. The Scientific Advisory Board (SAB) and Medical Advisory Board (MAB) members consist of leading scientists and clinicians drawn from the Rett syndrome field, augmented by distinguished neuroscientists and prominent individuals with experience in drug discovery and development within academia and industry.