Advancing Research.

Building programs to maximize our girls’ abilities (Neuro-Habilitation)

September 29, 2014

We’ve just announced some of the exciting studies in a brand new research area here at, and we wanted to take a moment to discuss what the program is and what aims to achieve. 


The Neuro-Habilitation Program.

Is a research program is aimed at maximizing function and abilities in the boys and girls with Rett syndrome. Targeting research for cognitive therapies, physical therapies, occupational therapies and speech therapies, that will directly impact them.

Recently, we held a special call for proposals, asking scientists, clinicians, therapists to bring us their best ideas that fit in this new Neuro-Habilitation Program. We specifically asked for research in the following areas: occupational therapy, physical therapy, communication; early diagnosis, early detection, and early intervention; or outcome measures for clinical research.


Why are we setting up this new program now? Why we are targeting these areas of research?

For over a decade now, we have put a significant amount of money into basic discovery research. Due to these groundbreaking studies, possible therapeutic targets for pharmacological (or drug) invention were discovered. Today, we support 2 human clinical trials and while these 2 drugs are still under clinical research and not yet an approved medication for Rett syndrome, it’s time to think ahead.

“He who fails to plan is planning to fail" …Winston Churchill

What happens after the girls are given a drug?  We need to prepare ourselves for the long road ahead to full habilitation. How are we going to support our girls in the near future? That means identifying new technologies and therapies that “reset the neurology” in Rett syndrome. We plan to be ready for pharmacological treatments that “correct Rett biology”.


We will award HeART grants to research projects that answer the call and were deemed of high scientific merit by peer reviewers Research Activities


This program aims to learn about or create technology which “resets the neurology”. Projects funded by us will receive a HeART grant because they “Help Accelerate Rett Therapeutics” (HeART). When we say “Therapeutics”, we think about pharmacological medication, which include therapies that enhance neuronal networks that maximize the abilities of our girls and boys.

So what does “reset the neurology” and “correct the biology” mean exactly?

We have to fix the connections of the brain. Think of an electrical circuit that needs to be wired correctly in order to turn on a light bulb. In terms of correcting the biology, we refer to pharmacological medications. These will help create new connections – by adding new wires and/or rewire existing wires.

But just as important, we need to reset the neurology and make sure those new circuits will fire to turn on that light bulb, or in the case of RTT, maybe stop the hand wringing, improve communication, speech, gait, etc… The latter is what Neuro-Habilitation is all about – to gain and improve ability and function.

The slide above highlights the Research Activities of Research, and indicates general approaches to reset the correct the biology and reset the neurology.


Who is driving the projects?

We reached out to all our networks to find interested researchers doing exciting work. This year we are pleased to support 5 grants that fall in the general areas of communication, occupational and physical therapies, and cognitive assessment.

Please visit for a brief summary of the funded projects.

~The Takeaway~

The Neuro-Habilitation Program identifies the best cognitive therapies, physical therapies, occupational therapies and speech therapies that aid in establishing the networks critical for skill development in Rett syndrome.

By conducting research on these two fronts – pharmacologic treatments and neuro-habilitation therapies, our hope is that the work will converge on each other, and we can match the best practices in drug therapy to the best practices in neuro-habilitation therapy, leading to the fastest way to improve the quality of life for those suffering with Rett syndrome.

The time is right to begin looking at this area of research.

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