Clinical Trial News & Updates

The most up-to-date information on clinical research to deliver treatments and cures for Rett.

More than 20 companies are currently working on treatments that will bring us even closer to a world without Rett syndrome. Fifteen years ago, there were none. Scroll down to learn more about the companies investing in Rett syndrome studies, the promising compounds being tested, and the most up-to-date status on their progress. For questions, please email us.


rett CLINICAL programs PIPELINE

Click the image below for an overview of all companies actively investing in Rett syndrome treatments. For more detailed information about each company’s progress, scroll down.

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Stages of clinical development

 The therapy development path is divided into five stages. To learn more about the research and treatment development process, visit our Rett Research Ready resource.

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How Are New Therapies Developed?


development and clinical trial status

Learn more about the companies investing in Rett syndrome below and the status of their development.

STAGE 5: REGULATORY APPROVAL & POST-MARKET SAFETY MONITORING

The following companies have successfully completed clinical trials and received regulatory approval for a Rett syndrome treatment.

STAGE 4: DRUG REVIEW

The following companies have applied to a regulatory agency like the FDA for permission to market a treatment after clinical trial results indicated the treatment is safe and effective. This stage may take 1-2 years once an NDA is submitted.

STAGE 3: ACTIVE CLINICAL TRIALS & RESEARCH 

The following companies are actively conducting clinical trials in individuals with Rett syndrome. This stage may take 6-7 years to complete the 3 phases of clinical trials depending on factors including participant recruitment, length of patient monitoring, and the clarity of results.

STAGE 2: PRE-CLINICAL RESEARCH & DEVELOPMENT

The following companies have a treatment that showed promise during the discovery stage, and is now being more vigorously tested in cell cultures and animal models following regulatory agency mandated requirements. This process lasts 3-5 years and if successful, results in an IND application to begin clinical trials.

STAGE 1: DRUG DISCOVERY & DEVELOPMENT

The following companies are in the discovery and development stage spanning basic research discoveries in the lab to tests in cell culture or animal models. This stage may include 5-10 years of testing before a new treatment can be developed further.

DISCONTINUED OR CANCELED CLINICAL PROGRAMS

The following companies have terminated their Rett syndrome treatment development program.

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