The Rett Syndrome Research Trust (RSRT) is the world leader in spurring and supporting scientific research on Rett Syndrome. The RSRT was launched in 2008 to drive research toward a cure for Rett Syndrome and related MECP2 disorders. Their mission is urgent: 350,000 girls and women globally live with the agonising symptoms of Rett Syndrome.  RSRT funds more research than any other Rett organisation in the US and abroad. Since 2008, RSRT has awarded $47 million to research. An average of ninety-six percent of every dollar donated to RSRT goes directly to their research program. Keeping their internal team lean and fiercely motivated allows them to focus funds and energy on what matters most: healing our children as quickly as possible.

As one of the few neurological disorders to show dramatic symptom reversal in mice, Rett Syndrome has attracted unprecedented interest from university and pharmaceutical scientists. Through innovative research and constant engagement with scientists, clinicians, industry, investors and affected families, the RSRT is at the epicenter of this activity.  RSRT do more than just write checks: they are changing the landscape of Rett research. They set the research agenda by proactively identifying and monitoring promising therapeutic areas, seeking out scientific and industry partnerships and working closely with them to advance programs through the drug development pipeline. RSRT believe that Rett Syndrome can be the first neurological disorder that can be cured. Their ultimate goal is to become obsolete – by finding that cure.

In March of 2017, the RSRT announced their bold strategic research plan called Roadmap to a Cure. The plan, which has total costs of $33 million over three years, includes four components:

RSRT will not be satisfied with subtle symptom improvement.

We want a cure.


Four priority approaches designed to fix the underlying cause of Rett Syndrome are being pursued: 1) Gene therapy, 2) MECP2 Reactivation, 3) Protein Replacement, and 4) RNA Modifications. Informed by critical data generated by RSRT-supported researchers, the biotech company AveXis is now advancing the lead MECP2 gene therapy candidate toward clinical trials. This will be the first clinical trial of a potentially curative approach for Rett Syndrome. This is a key step towards RSRT delivering on its goal. Funding needed: $13 million.


Until a cure is achieved, RSRT will pursue treatment strategies with the greatest potential to improve quality of life for those suffering with Rett Syndrome. Therapeutic interventions may improve multiple symptoms. Funding needed: $3.3 million.


There are currently no clear outcome measures for clinical trials in Rett Syndrome. Working with the Rett community, scientists, and clinicians, RSRT is establishing outcome measures that can be used by the FDA and other regulatory agencies. This will shorten the timeline for the testing and approval of potential cures and enable pharmaceutical and biotech industry investment. RSRT is also putting significant resources into providing clinics with the resources and personnel they will need to implement the most effective and efficient clinical trials. Funding needed: $9.2 million.


RSRT must continue investing in basic science to expand their understanding of the mutated gene that causes Rett Syndrome, its protein product, and its function. This is vital to informing all approaches.  Funding needed: $4.5 million.

RSRT has exceptionally low overhead expenses. Operational costs for Roadmap to a Cure are $3 million, less than 10% of the total budget.

Watch the RSRT’s Roadmap to a Cure

In June of 2017, biotech company AveXis announced that based on encouraging data generated by RSRT’s Gene Therapy Consortium, it would advance the lead MECP2 gene therapy candidate AVXS-201 towards clinical trials within a year, pending FDA approval.  To learn more please visit

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