IRSF Research
IRSF Research
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.
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
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
Research that’s already changed the game.
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
The IRSF Approach
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
But we can’t do it without you. Here are the best ways to help:
But we can’t do it without you. Here are the best ways to help:
Donate
Participate in a study
Advocacy
News and Research
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
Seals of Approval
