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? Join us.

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.
Discovery of MECP2
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.
Discovery of Rett Reversal

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.

First Clinical Trial for Rett

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.

Critical Infrastructure

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.

Involving Families

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.

Companies Investing in Rett Syndrome Treatments

More than 20 pharmaceutical companies are currently working on treatments for Rett.
Fifteen years ago, there were none.

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 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:

Voice of the Patient Report Now Available

A summary of the Externally-Led Patient-Focused Drug Development Meeting held on Rett syndrome in March 2022 is now available as a reference document to the FDA and pharmaceutical companies developing treatments for Rett syndrome. 

FDA Approves Trofinetide for Treatment of Rett Syndrome

On March 10, 2023, Acadia Pharmaceuticals announced that its investigational drug, Trofinetide, was approved by the FDA. The drug, marketed in the US under the name DAYBUE, becomes the first-ever FDA-approved treatment for Rett syndrome.

First Rett Syndrome Gene Therapy Trial in U.S. Launches

Neurogene announced the locations, and protocol, and began enrolling for the first U.S. Phase 1/2 clinical trial of their investigational gene therapy, NGN-401. The clinical trial will enroll 5 females with Rett syndrome aged 4-10 at 3 locations. 

Research Collaborators

Neuren Pharmaceuticals

Seals of Approval

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