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