Accelerating Research: "We are in the Time of Rett"


We are living in an exciting and hopeful time - “the time of Rett” - where finding a treatment and a cure for Rett is truly possible.

Today, we want to share HOW committed we are to the work scientists and clinicians are doing to find treatments and a cure for Rett syndrome.

Our Scientific Strategy  is a ‘full spectrum’ research approach.
We plan to accelerate research and aggressively move more potential drug treatments to clinical trials, while developing neuro-habilitation programs that address the immediate needs of those affected by Rett syndrome.

To date invested $35 million to research in many programs including:

  • Discovery research to generate new ideas
  • Translational research to turn new ideas into medicine and treatment
  • Clinical research to turn medicines and treatments into best practices

We have partnered with leading Rett clinicians and clinic centers for Rett syndrome, MECP2 Duplications, and Rett related disorder's Natural History Study.

We have implemented a foundation-driven drug-screening program that is based on the key biology that needs to be targeted for treatment called the Scout Program.

We have supported successful early stage human clinical research trials that will move into Phase 3 studies.

We have launched the Neuro-Habilitation Program to develop cognitive therapies, physical therapies, occupational therapies and speech therapies for skill development in Rett syndrome.

Please watch this video where Chief Science Officer Dr. Steve Kaminsky explains why our research strategy has multiple approaches to finding scientific solutions to reverse and modify the complex symptoms of Rett syndrome.

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If you are interested in reading more about Neuro-Habilitation, our program was inspired by the following books:

  • Soft Wired -Micheal Merzenich
  • The Brain That Changes Itself - Norman Doidge
  • Spark: The Revolutionary New Science of Exercise and the Brain - John Ratey
  • Remarkable Discoveries and Recoveries from the Frontiers of Neuroplasticity - Norman Doidge.