Our Research

All in at every level

Much goes in to creating treatments and cures for Rett syndrome. IRSF’s investments have already produced game-changing discoveries and built essential treatment development infrastructure. Our leadership and support direct Rett research along a path to treatment and cures for all individuals with Rett.

Our strategy

IRSF’s Scientific Strategy consists of three guiding pillars that cover Rett research at every level:


Critical research
– Critical research addresses gaps in researchers’ knowledge of Rett syndrome, including MECP2 and other targets that give rise to the pathology of Rett. It equips researchers with the information they need to devise new ways to treat Rett and improve upon existing ones.



Pipeline development
– The Rett treatment development pipeline represents the assessments potential new treatments undergo to prepare for testing in clinical trials. IRSF works to improve the pipeline while helping companies developing Rett treatments to navigate it.



Clinical trials
– IRSF supports Rett clinical trials through facilitating communication and connection among the clinical trial stakeholders: Rett families, physicians, the FDA, and trial sponsors. At the same time, it invests in the infrastructure necessary for successful clinical trials such as Rett registries and research onoutcomes measures.

Rett Syndrome Centers of Excellence

We partner with 22 Rett syndrome clinics across North America to provide care and treatment to those with Rett syndrome and participate in our North American Rett syndrome Clinics Network. The International Rett Syndrome Foundation has created an innovative clinic program designation, the IRSF Centers of Excellence for US clinics that meet rigorous criteria and provide exceptional care to those with Rett syndrome.

2022 Center of Excellence Application

IRSF Center of Excellence Network
University of Alabama Birmingham Civitan International Research Center, Birmingham, AL
Children’s Hospital Los Angeles, Los Angeles, 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
Kennedy Krieger Institute, Baltimore, MD
Boston Children’s Hospital and Harvard Medical School, Boston, MA
Gillette Children’s Specialty Healthcare, St. Paul, MN
Washington University School of Medicine and St. Louis Children’s Hospital, St. Louis, MO
Children’s Hospital at Montefiore, Bronx, NY
The University of North Carolina at Chapel Hill, Chapel Hill, NC
Cincinnati Children’s Hospital, Cincinnati, OH
Nationwide Children’s Hospital, Columbus, OH
Children’s Hospital of Philadelphia, Philadelphia, PA
Greenwood Genetic Center, Greenwood, SC
Vanderbilt University School of Medicine and Vanderbilt Kennedy Center, Nashville, TN
Texas Children’s Hospital, Houston, TX
University of Texas Southwestern with Children’s Health Dallas, Dallas, TX