IRSF Collaboration Opportunities For Industry

Our Foundation has the experience and tools you need to accelerate your Rett syndrome treatment development program.

IRSF has established resources to facilitate every phase of Rett syndrome treatment development. We offer them to industry for research that aligns with our goals. Let’s work together to address the unmet need for treatments for Rett syndrome, a genetic neurodevelopmental disorder that causes severe disabilities in children and impacts every aspect of their lives.

Learn more about how we support our industry partners. Contact us.

Why Rett? | Therapeutic Targets | Our Resources | Why IRSF? | Contact Us

“We had very useful discussions with [IRSF]. It was particularly helpful to speak to those who have seen what the data looks like through these stage processes with other compounds.” (on working with IRSF through their Scout Program for Rett syndrome drug testing)
Mark Carlton, PhD, Cerevance CSO
“Thank you to the International Rett Syndrome Foundation (IRSF) and the entire Rett community who help make this research possible. We appreciate your continued partnership on this journey to find a treatment to address the core symptoms of Rett syndrome.”
Acadia discussing IRSF’s support of their Trofinetide clinic
“We are delighted to continue our partnership with and its network of experts to accelerate the clinical development of ANAVEX 2-73 for the treatment of Rett syndrome,” 
Christopher U. Missling, PhD, President and Chief Executive


Rett syndrome is a rare disorder that affects 1 in every 10,000 children worldwide. The disorder typically appears between 6 and 18 months of age and dramatically increase in severity in early childhood. It causes impairments that impact almost all aspects of an individual and their family members’ lives. There are currently no approved treatments for Rett syndrome. However, we believe that life-altering treatments are within reach because Rett syndrome has:

  • One Causal Gene
  • A Predictive Mouse Model
  • 15 Years of Longitudinal Natural History Data
  • Established Centers of Excellence
  • Clinical Endpoints in Development

Rett syndrome



Research has identified several targets and approaches that show promise for treating Rett syndrome. These include:

  • MeCP2
  • GABA
  • Glutamate
  • BDNF
  • NGF
  • IGF-1
  • Sigma-1 Receptor
  • Microglia


As the leading Rett syndrome research and advocacy organization, the International Rett Syndrome Foundation (IRSF) builds upon our nearly 40-year commitment to breakthrough discoveries and life-changing advancements in research toward a cure while supporting families affected by Rett syndrome.

Through our legacy foundation pioneers, we have invested over $54M in research leading to identifying Rett syndrome’s cause, proving Rett syndrome is reversible in mice and developing multiple clinical trials for Rett syndrome. We fight for families living with Rett syndrome and a world without it.

Our Highlights:

  • Funded the discovery of the gene that causes Rett syndrome (MECP2)
  • Funded the discovery of reversibility of Rett syndrome in mice
  • Funded early gene therapy research in Rett syndrome
  • Funded the first Rett syndrome clinical trial
  • Funded the development of the first Phase 3 clinical trial
  • Convened parents and experts to publish the Rett Syndrome Handbook
  • Designated 15 Rett Syndrome Centers of Excellence

Companies We’ve Worked With


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We have our finger on the pulse of all relevant emerging science and technologies, and we won’t stop until we have treatment options for all those living with Rett syndrome. We’re ready to share our knowledge and experience with those who share our goals. Contact us to get started.