Rett Syndrome Treatment

Since no two individuals with Rett are exactly the same, each one needs a personalized treatment plan. Our resources help clinicians provide the best care possible for their patients with Rett.

Treating Rett Syndrome

Any treatment decision should be made based on the individual and family’s needs, goals, and values in consultation with a healthcare provider. IRSF provides clinicians with actionable resources to deliver the highest quality of care.

Comprehensive Care Guidelines

The IRSF Rett Syndrome Comprehensive Care Guidelines and Quick Reference Guide were developed in collaboration with Rett syndrome experts throughout the U.S. These guidelines help support a meaningful partnership between the caregivers, physicians, nurses, and therapists involved in the care of individuals with Rett syndrome. The guidelines include a Rett-related review of systems, physical exam findings, and recommended screening tests.

IRSF would like to express gratitude to the 18 Rett syndrome Centers of Excellence and experts who volunteered time and effort to develop this list of recommendations.

Rett Expert Clinical Collaboration Group

Do you have questions about the clinical management of Rett syndrome? The Rett Expert Clinical Collaboration Group provides a space to engage with leading Rett syndrome experts and access resources specifically for clinicians treating individuals with Rett. The group is moderated by experts from our Center of Excellence Network with expertise in neurology, complex care, gastroenterology, epileptology, nursing, and more.

The First and Only
FDA-Approved
Treatment for Rett Syndrome

DAYBUE™ (trofinetide) is a prescription medication taken orally to treat Rett syndrome in adults and children 2 years of age and older. In the LAVENDER™ trial, doctors and caregivers reported improvements in the signs and symptoms of Rett syndrome at 12 weeks compared to placebo. DAYBUE is not a cure for Rett syndrome.

Rett Community Impact

Trofinetide: From Discovery to FDA Approval

The path to FDA approval of DAYBUE™ (trofinetide) for the treatment of Rett syndrome was more than a decade in the making. Investing nearly $2M, IRSF worked in close collaboration with industry leaders and the Rett community to reach this groundbreaking milestone, developing the first FDA approved treatment for Rett syndrome.

2012

Dr. Steve Kaminsky, IRSF's then-CSO, identifies the potential benefit of the compound for Rett syndrome and connects with leaders at Neuren Pharmaceuticals for further investigation.

2013

Neuren begins an adult Phase 2 clinical trial of Trofinetide (then called NNZ-2566), with nearly $800K of funding from IRSF.

2016

Neuren begins an adult Phase 2 clinical trial of Trofinetide (then called NNZ-2566), with nearly $800K of funding from IRSF.

2018

Acadia Pharmaceuticals and Neuren enter into a North American License Agreement for the continued development and commercialization of Trofinetide.

2019

Acadia begins the Phase 3 LAVENDER study in girls aged 5-20 with support from IRSF to identify clinical trial sites and promote participant recruitment.

2022

Acadia submits a New Drug Application (NDA) to the FDA. The NDA is accepted and granted priority review.

March 2023

Trofinetide, now called DAYBUE™, becomes the first FDA-approved treatment for Rett syndrome!

Additional Resources

IRSF makes it easy to find and enroll patients in clinical trials for Rett syndrome. We also offer valuable patient care resources for healthcare providers involved in the treatment of an individual with Rett syndrome.

Find a Clinical Trial

myRett Trial Finder

Find Patient Care Resources