Research Blogs






Rett Syndrome Gene Therapy:  Understanding the Published Data

Steve Kaminsky, PhD, Chief Science Officer and Janice Ascano, PhD, Manager of Grants and Research,,  July, 2017

Understanding the status of gene therapy research for Rett syndrome and our review of the five published papers on gene therapy.

Neuro-Habilitation Research - Building programs to maximize abilities

Steve Kaminsky, PhD, Chief Science Officer, June, 2017

What is the Neuro-Habilitation Program? What is Neuro-Habilitation Research? Why we are targeting Neuro-Habilitation research? Who is driving the projects?

The challenges of assessing cognitive function in children with Rett Syndrome 

Charles A. Nelson & Lauren Baczewski

Participating in standard cognitive testing can be challenging for those with Rett Syndrome (RTT) due to the disorder’s impact on language and motor skills. Our goal is to minimize verbal and motor requirements to obtain a more objective reading of the child’s cognitive ability by adapting the assessment to be administered on an eye tracking computer.  In doing so, we rely on the child’s ability to engage in coordinated eye movements to illustrate their answer to each question.

Research Strategy Blog- What is “Full-Spectrum Research”?

Steve Kaminsky, PhD, Chief Science Officer, April, 2017

What is ‘full spectrum’ research and how does it differentiate It is identifying the best genetic and pharmacologic research to correct the biology of the brain’s neurons, while also studying how to reset the neurology - the neural pathway development- through physical, occupational, speech and cognitive therapies.

Clinical Trials: Understanding their importance and how to participate

Steve Kaminsky, PhD, Chief Science Officer, January 2017

Click here to read Dr. K's blog answering your most pressing questions about clinical trials


An update from our Chief Science Officer

Steve Kaminsky, PhD, Chief Science Officer Rettsyndrome.orgFebruary 5, 2016

As we begin 2016 our research strategy remains consistent – to cover the full spectrum of Rett syndrome research from basic discovery, where ideas begin, to translating discoveries into medicines for future clinical testing and treatments.


Impatient Revolution:  A guide for impatient patient organizations

Ana Mingorance, PhD, Dracaena Consulting, April 5, 2017

Read Dr. Mingorance's perspective on non-profit fundraising organizations, their research, and their clinical trials.


Trofinetide for Rett Syndrome in Recent News

Public release: June 22, 2015

A novel agent is holding out some much-needed promise for girls and women with Rett syndrome.


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

Steve Kaminsky, PhD, Chief Science Officer

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.