Rett Syndrome may become the first reversible childhood neurological disorder. It is the goal of the Trust to deliver clinical trials of a novel treatment strategy that addresses the underlying genetic defect within five years.

To achieve this goal, RSRT ventures beyond the traditional scope of disease-specific foundations. The Trust will operate as a virtual biotechnology company outsourcing the various drug development steps to the best qualified academic institutions or contract research organizations.

In keeping with our time-sensitive mission, strategies identified as the most readily translatable into new treatments are being pursued in parallel. The Trust will quickly prioritize resources as new modalities emerge. 

The Trust is the catalyst for a private sector effort to bridge the critical funding gap that exists between basic research financed by the government and later-stage drug development undertaken by pharmaceutical companies and biotech.

Without a privately financed effort, treatments and a cure for Rett Syndrome will remain many decades away.

Once a drug(s) or biological(s) emerges, RSRT will either partner with industry or establish its own biotech company to bring the treatment(s) to the clinic.

The Trust brings valuable resources to the Rett research community:

• deep knowledge of the disease
• emphasis on translational effort
• prioritization of research projects
• financial resources for high-impact science
• global clearinghouse of information
• access to intellectual capital, tools and reagents
• connections to the patient community
• project management capabilities
• scientific meetings with focused topics on an as-needed basis.

Banner image - Neurons in the dentate gyrus (part of the hippocampus) of a genetically modified "brainbow mouse". The image is taken using a confocal microscope. 
Photograph courtesy of Jean Livet/Nature