Help for Today, Hope for Tomorrow.
The International Rett Syndrome Foundation (IRSF) is the leading research, family support, and advocacy organization for Rett syndrome. We fund trailblazing research seeking treatments and a cure, empowering support that helps families cope and offers hope, and increasing awareness of this rare disorder that can affect nearly every aspect of any child’s life.
At IRSF, we are transforming lives every day in our fight to treat and cure Rett syndrome, presenting the best opportunity to better the lives of families living with this disorder.
Trailblazing Research
We have a track record of delivering real results: our highly comprehensive scientific program—bolstered by unprecedented industry partnerships—is the only one to include advancing genetic approaches, developing drug treatments, clinical research, and more.
Empowering Families
We connect with and support families every single day to provide easy steps and tools to help them thrive—including building a strong community of parents and experts that help generate key insights that, in turn, inform research.
Compelling Advocacy
We advocate for policies and funding that improve care, expand support, and advance research for the Rett community. Through our relentless efforts, we drive meaningful change to ensure better resources, services, and outcomes.
What is Rett Syndrome?
Rett syndrome is a rare and devastating neurodevelopmental disorder that impacts nearly every aspect of an individual’s life, including their ability to speak, walk, eat, and even breathe. Rett is a genetic disorder caused by a spontaneous pathogenic variant in the MECP2 gene. When the MECP2 gene does not function properly, it can cause issues throughout the entire body, leaving individuals understanding more than they can communicate.
Every individual with Rett is unique and so are their symptoms which begin as early as 6 months old when parents see their child miss development milestones and lose abilities they had already gained. Though Rett occurs most often in females (approximately 1 in 10,000 births), boys can have Rett too.
You are not alone.
Receiving a Rett syndrome diagnosis can feel overwhelming. At IRSF, we are unwavering in our commitment to helping your family meet the demands of life with Rett—today and tomorrow. We create in-depth resources, education, and community connections that elevate parents and caregivers into champions for themselves, their loved ones living with Rett syndrome, and the broader community.
IRSF Center of Excellence Clinic Network
Find expert care and treatment through our trusted network of best-in-class clinical and research centers.
Rett Research & Treatment Pipeline
Discover the latest on Rett syndrome research and the progress of the companies working to treat and cure the disorder.
Rett Education Programs
Check out our latest RettEd programs including educational opportunities, events, and more designed to help families connect and thrive.
Together, we can create a world without Rett syndrome.
Our understanding of how Rett syndrome works and how to successfully treat it is advancing more rapidly than ever before. Treatments and cures are not an if, but a when. Join us in giving hope to families all over the world living with Rett.
Ways to Give
Your support makes a difference. On average, more than 80 cents of every dollar donated supports our mission.
Fundraise for Rett
Start an online fundraiser, participate in an annual IRSF event, or start one of your own.
Raise Awareness
Help spread the word and raise awareness about Rett syndrome.
Advocate for Rett
Stand with us in advocating for policies and funding that benefit those with Rett syndrome.
Community Events & More
Join us at events and initiatives that support the Rett syndrome community.
Discover the information you need on Rett Syndrome.
IRSF offers resources to support the entire Rett community in advancing treatments, care, and understanding of Rett syndrome. Find out more about what we have to offer by choosing a category below.
Rett Syndrome News & Updates
Stay informed on the latest developments in Rett syndrome research, treatments, and community news.
Raising a Hand: A Journey of Healing, Hope, and Friendship
DAYBUE in the Real World
Answers to your most frequently asked questions and resources to find out more about the first-ever FDA-approved treatment of Rett syndrome. According to the National Organization for Rare Disorders (NORD), […]
Transforming the Landscape of Rett Research
The International Rett Syndrome Foundation (IRSF) hosted its annual scientific meeting in Westminster, Colorado on June 18-19, 2024, as part of its ASCEND 2024 Rett Syndrome National Summit. By Mackenzie […]
Spotlight on Communication: An Interview with Dr. Theresa Bartolotta
For Theresa Bartolotta, Ph.D., CCC-SLP, good things have come through community. Bartolotta, a speech-language pathologist (SLP) with more than 40 years of clinical experience, discovered her passion for helping others […]
Advocating for YOU: Rare Disease Week on Capitol Hill
IRSF works tirelessly to advocate for you and your loved one with Rett syndrome. Our work takes many forms, our persistence is often unseen, and our successes are the result […]
Neurogene Announces Expansion of Gene Therapy Clinical Trial
Today, Neurogene announced plans to expand and more rapidly enroll patients their Phase 1/2 clinical trial of investigational gene therapy NGN-401. For the full press release, click here. Key Facts: […]
Taysha Gene Therapies Adolescent & Adult Study Expanded to U.S.
Today, Taysha Gene Therapies shared several exciting updates on their REVEAL Phase 1/2 clinical trial of investigational gene therapy TSHA-102: The study will now expand to include adolescent and adult […]
My Rett Ally: Revolutionizing Care Management for Rett Families
On a recent visit to the emergency room, it took just seconds for Samantha Brant to pull up a detailed, up-to-date medication list for her daughter, Macy. Macy lives with […]
Taysha Gene Therapies Doses First Pediatric Patient in U.S. Gene Therapy Clinical Trial
Taysha Gene Therapies today announced the dosing of the first pediatric patient in their REVEAL Phase 1/2 trial of investigational gene therapy TSHA-102. For Taysha’s full press release, click here. […]
Neurogene Announces First Pediatric Patients Dosed in US Gene Therapy Clinical Trial
We’re excited to share that today Neurogene announced that two pediatric patients have been dosed in their Phase 1/2 trial of investigational gene therapy NGN-401. For Neurogene’s full press release, […]
Taysha Gene Therapies Updates Community on Positive Initial Findings and Expansion of TSHA-102 Clinical Trial
Today, Taysha Gene Therapies made several exciting announcements about the progress of their REVEAL Phase 1/2 trial in Canada and the future study of their gene therapy, TSHA-102. To read […]
Acadia Pharmaceuticals Acquires International Rights to Trofinetide
BIG NEWS for our international Rett syndrome community! Today, Acadia Pharmaceuticals announced they have acquired the rights to make trofinetide available for the treatment of Rett syndrome across the globe. […]
RettAway Reflections
By Samantha Brant, Family & Community Engagement Manager, and mom to Macy As I returned home from this year’s RettAway at Morgan’s Inspiration Island, my heart swelled with not only […]
Clinical Trial Updates from Taysha & Anavex
We are excited to share news from two pharmaceutical companies working on potential treatments and cures for Rett syndrome. Taysha Gene Therapies Taysha Gene Therapies shared that they are encouraged […]
Neurogene Announces Locations & Opens Enrollment for US Gene Therapy Clinical Trial
We’re excited to share that today Neurogene announced the locations and began enrolling for the first U.S. Phase 1/2 clinical trial of their investigational gene therapy, NGN-401. Enrollment at Texas […]
A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!
This first-ever treatment is approved for individuals with Rett syndrome. (Updated March 19, 2023) On March 10, 2023, Acadia Pharmaceuticals announced that their investigational drug, Trofinetide, has been approved by […]
Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.
We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug Application (IND) to launch a clinical trial for their gene therapy treatment NGN-401 in […]
Flu, COVID, & RSV: A Message to our Rett Community
By Dr. Tim Benke, IRSF Medical Advisor As many of you know, the United States is currently facing the triple impact of flu, RSV, and COVID-19. These respiratory illnesses are […]
Taysha Begins Recruitment for Gene Therapy Trial
Next Huge Milestone for Rett The last 12 months have been an unprecedented time for Rett syndrome. In December 2021, Acadia Pharmaceuticals reported that the first (and only) Phase 3 […]
Your Voice Matters to the FDA & Pharmaceutical Industry
By Dr. Dominique Pichard, IRSF CSO – The patient voice has increasingly become a focus for the Food and Drug Administration (FDA) in the drug development process. Thanks to years […]
FDA Grants Trofinetide Priority Review
ACADIA has shared the exciting news that the US Food and Drug Administration (FDA) has reviewed the documents received as part of the New Drug Application (NDA) for trofinetide, and […]
In-Person Strollathons Are Back!
In September IRSF returns with our in-person Strollathons! These nationwide fundraisers bring together local communities to go ALL IN for treatments and a cure for Rett syndrome. Since 2004, these […]
IRSF Expands Center of Excellence Network
The International Rett Syndrome Foundation is expanding our community’s access to best-in-class care by adding four Rett syndrome clinics to our Center of Excellence network. This expansion helps to fulfill […]
The Summit of Hope and Healing: ASCEND 2022 Rett Syndrome National Summit
By Katie Busch, IRSF Program Coordinator – During the last week of April 2022, the International Rett Syndrome Foundation welcomed more than 500 members of the Rett community to the […]
Accelerating Discoveries in Rett Research: 2022 IRSF Rett Syndrome Scientific Meeting
In April 2022, the International Rett Syndrome Foundation hosted the IRSF Rett Syndrome Scientific Meeting in advance of the foundation’s first in-person family summit in 6 years. The 2-day meeting […]
Taysha Rett Syndrome Community Update
From IRSF: 2022 is a big year for Rett syndrome: the return of an in-person Family Summit, a successful EL-PFDD meeting to share the voice of the community with the […]
IRSF Sets Up Next Wave of Rett Breakthroughs with $2.3 M Research Investment
Dr. Dominique Pichard, IRSF Chief Science Officer Get ready for the next wave of research targeting treatments and cures for Rett syndrome. IRSF is making it happen. “Because Rett syndrome […]
Positive Top-line Results for Phase 3 Trofinetide Trial
Melissa Kennedy, Chief Executive Officer On December 6, the Rett syndrome community received some very encouraging news: Acadia Pharmaceuticals reported that top-line results for its Lavender Phase 3 clinical trial for […]
Help Unlock Treatments & Cures for Rett with IRSF’s Rett Syndrome Registry
Your family can be the key that researchers and industry need to create a world without Rett. And the International Rett Syndrome Foundation (IRSF) is making it easier than ever […]
International Rett Syndrome Foundation’s New Center of Excellence Network
The International Rett Syndrome Foundation is committed to advancing and expanding our community’s access to best-in-class Rett syndrome care. Toward that end, we are designating 15 partner clinics across the […]
Kelsey & Toyua: Friendship & Hope Just 20 Minutes Away
Kelsey Clark felt alone and isolated when her five-year-old daughter Charlee was diagnosed with Rett syndrome in April 2019. Kelsey, her partner Jordan, Charlee, and their two-year-old son Bennett live […]
Board of Directors Welcomes Alan Shukovsky —The Father of a Boy Living With Rett
When Alan Shukovsky and his wife, Eva, were getting married, their rabbi told them that it was really important for their family to find a personal mission—a cause—they could support […]
Give them the opportunity for a brighter future.
Your support helps provide families with the resources they need while advancing the search for better care, treatments, and someday a cure. Join us in improving lives and fostering hope for individuals impacted by Rett syndrome.