IRSF Research

Finding Solutions for all

IRSF Research

Finding Solutions for all

Rett syndrome is a
complex disorder that
manifests in many ways.

Rett syndrome is a complex disorder that manifests in many ways.

A one-size-fits-all approach would leave families around the world without hope. Through the support of our donors, IRSF has been investing in projects that impact foundational Rett syndrome research.
A one-size-fits-all approach would leave families around the world without hope. Through the support of our donors, IRSF has been investing in projects that impact foundational Rett syndrome research.
At the same time, we empower Rett families to get involved as partners in the research that may one day save their loved one’s life.
Our strategy is simple, but powerful: improve care today and create treatments for tomorrow. Because every family fighting Rett deserves hope.

We’re going all in for a cure no matter what it takes.

Are you a researcher or a pharmaceutical company?

Click to see how we can partner with you to make a difference for Rett.

Testimonials from our friends in the scientific community

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 PHARMACEUTICALS

What [the people that IRSF supports] are doing is moving the bar forward. You don’t know where the ‘aha’ is going to come from. But if you don’t search, you’re never going to find the answer.

Kerry Delaney
PhD, University of Victoria

Research that’s already changed the game.

IRSF research has laid a foundation for scientific breakthroughs and clinical trial successes.

We funded research that made the foundational discovery of which gene causes Rett syndrome: MECP2. This knowledge changed our understanding of Rett and provided researchers with the information they need to devise potential treatments.

We funded critical research that discovered Rett could actually be reversed. This discovery continues to inspire researchers to work on Rett in the hope of finding cures.

The first-ever placebo-controlled clinical trial for a treatment specifically for the underlying disorder of Rett syndrome was funded by IRSF. Clinical trials are a critical step in testing a treatment in the desired population (those with Rett) in order to determine if they work — and can become FDA-approved treatments.

You can’t travel efficiently from one city to another without a road, and Rett treatment research can’t advance effectively without a standardized path. Our involvement with initiatives such as the Natural History Study, the Kaminsky Scout Program, and IRSF Centers of Excellence has helped create and improve such a path.

Treatment development works faster and better when the family's needs are kept in focus. Our leadership initiatives such as Rett Research Ready and Rett Syndrome Registry have placed families front and center in the process of developing treatments for Rett.

The IRSF Approach

We work tirelessly to approach Rett solutions from several strategic angles, creating a robust pipeline of research, drug development and clinical trials. Your support makes it possible to fund game-changing discoveries, build a robust research pipeline, and break down barriers to clinical trials.

The IRSF Approach

We work tirelessly to approach Rett solutions from several strategic angles, creating a robust pipeline of research, drug development and clinical trials. Your support makes it possible to fund game-changing discoveries, build a robust research pipeline, and break down barriers to clinical trials.

Help us create a world without Rett

As we relentlessly pursue a cure, we come alongside families with support, wherever they are on the journey.
But we can’t do it without you. Here are the best ways to help:
As we relentlessly pursue a cure, we come alongside families with support, wherever they are on the journey.
But we can’t do it without you. Here are the best ways to help:

Donate

We’re working to raise $29 Million dollars to go all in for every family and individual fighting Rett. With your donation, you can help us fund discoveries and be one step closer to finding a cure.

Participate in a study

Treatments and cures cannot become a reality without volunteers willing to participate in research. By joining a study, your family will help shape Rett research for current and future generations.

Advocacy

Major organizations like the NIH and the US Department of Defense can provide the large sums needed for big ticket Rett research essentials if they understand the need. Help us get Rett syndrome on their agenda.

News and Research

Keep up with the latest in Rett syndrome research including the clinical research pipeline:

IRSF Sets Up Next Wave of Rett Breakthroughs with $2.3 M Research Investment

Get ready for the next wave of research targeting treatments and cures for Rett syndrome. IRSF is making it happen.

Positive Top-line Results for Phase 3 Trofinetide Trial

Acadia Pharmaceuticals reported that top-line results for its Lavender Phase 3 clinical trial for trofinetide showed significant improvement over placebo.

Hive Networks & IRSF Partner to Advance Research and Treatment for Rett Syndrome

Hive Networks today announced a multi-year partnership with the International Rett Syndrome Foundation to provide a digital health platform to support the Rett community.

Research Collaborators

Neuren Pharmaceuticals

Seals of Approval

Scroll to Top