Our Research Strategy

We are accelerating Rett syndrome research toward clinical testing to create life-changing treatments and a cure for those diagnosed with Rett syndrome. We have invested over $49 million in research to find new discoveries, to translate ideas into medicine, to move those medicines into clinical trials, and to create therapies to help individuals regain the skills and abilities stolen by Rett syndrome.

We are committed to creating solutions for today, while we work to create
a world without Rett syndrome tomorrow.

Funded Research Spectrum

Graphic showing Rett Research Breakdown

Scientific Advisory Board

Dr. Huda Zoghbi, M.D., Director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, Professor at Baylor College of Medicine, and Investigator with the Howard Hughes Medical Institute
Dr. James Eubanks, Ph.D., Senior Scientist and Research Division Head at Krembil Research Institute, at the University Health Network in Toronto
Dr. Qiang Chang, Ph.D., Neuroscientist, and Director of the Waisman Center at the University of Wisconsin-Madison
Dr. Zhaolan (Joe) Zhou, Ph.D., Molecular geneticist and neuroscientist at the University of Pennsylvania, and Director of the Preclinical Models Core at the Intellectual and Developmental Disabilities Research Center at Children’s Hospital of Philadelphia
Dr. Joseph Ecker, Ph.D., Molecular biologist at the Salk Institute for Biological Studies, where he is the International Council Chair in Genetics, Director of the Genomic Analysis Laboratory, and an Investigator with the Howard Hughes Medical Institute
Dr. Kyle Fink, Ph.D., Neuroscientist at the Institute for Regenerative Cures at UCDavis
Dr. Alysson Muotri, Ph.D., Professor at the Sanford Consortium for Regenerative Medicine at UC San Diego
Dr. Haiyan Fu, Ph.D. Scientist at the Gene Therapy Center at the University of North Carolina
Dr. Sharad Verma, Ph.D., Program Director in the Preclinical Therapeutics Grants Branch (PTGB) at the National Cancer Institute (NCI) at the National Institutes of Health (NIH)
Dr. Marcie Glicksman, Ph.D., Neuroscientist and Head of biology at EnClear Therapies
Dr. Tiina Urv, Ph.D., Behavioral scientist in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS) at the NIH
Dr. Liz Berry-Kravis, M.D., Ph.D., Child neurologist and the Co-Director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush University

Clinical Research Centers of Excellence

Rettsyndrome.org has created an innovative clinic program designation, the Rettsyndrome.org Clinical Research Centers of Excellence. In North America, we work with 22 Rett syndrome clinics that provide care and treatment to those with Rett syndrome and participate in our North American Rett syndrome Clinics Network. We also recognize the 14 clinics that have been involved with the Natural History Study for their long commitment to studying and improving the lives of those who suffer from Rett syndrome as a Rettsyndrome.org Clinical Research Center of Excellence.


Rettsyndrome.org Clinical Research Centers of Excellence
University of Alabama Birmingham Civitan International Research Center, Birmingham, AL
University of California San Diego, Rady Children’s Hospital, San Diego, CA
University of California San Francisco Benioff Children’s Hospital Oakland, Walnut Creek, CA
Children’s Hospital Colorado, Aurora, CO
Rush University Medical Center, Chicago, IL
Boston Children’s Hospital and Harvard Medical School, Boston, MA
Gillette Children’s Specialty Healthcare, Saint Paul, MN
Washington University School of Medicine Saint Louis Children’s Hospital, Saint Louis, MO
University of Rochester Medical Center, Rochester, NY
Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
Children’s Hospital of Philadelphia, Philadelphia, PA
Greenwood Genetic Center, Greenwood, SC
Vanderbilt University School of Medicine and Vanderbilt Kennedy Center for Research on Human Development, Nashville, TN
Texas Children’s Hospital, The Blue Bird Circle Rett Center, Houston, TX

Communications Guidelines Pop-up