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We are the organization working to cure Rett Syndrome. As the largest worldwide funder of Rett research, we're advancing genetic medicines that target the root cause of Rett. The gene therapy programs currently in clinical trials were started and incubated at RSRT. We are the go-to partner for genetic medicine scientists and companies.

Our critical work is made possible by the commitment and passion of Rett families in the US and around the globe. If you want a cure as urgently as we do, we welcome you to our site and invite you to join us in our quest.

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Roadmap to Cures 2.0

Built on decades of scientific advances, Roadmap to Cures will select and drive innovation for three genetic medicines that attack the root cause of Rett syndrome, and advance into clinical trials by 2028.

Our goal is to put ourselves out of business as quickly as possible.

Recently Diagnosed?

Getting a Rett syndrome diagnosis is one of the hardest things you’ll ever face. We know this first-hand. Emotions can run the gamut and it can all seem overwhelming. You’re worried about your child and your family. You’re reading everything you can get your hands on but unsure how to interpret it or if it's relevant. We’ve been there, just reach out to us: we’ve got your back.

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Rett Syndrome Global Registry

Global Registry

Imagine the power of Rett parents worldwide aggregating their knowledge and sharing it with clinicians and biopharma while improving their ability to care for their own child. We are making it happen. #ParentPowered

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Meet Stuart

Meet Stuart Cobb, PhD. He started his career as a basic scientist, but once introduced to Rett he changed course to focus on developing genetic medicines. He is the scientist behind Neurogene’s Rett gene therapy program. If you’re looking for a way to support scientists like Dr. Cobb who are committed to curing Rett syndrome then join us in our quest.

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MECP2 Duplication

MECP2 Duplication syndrome (MDS) is caused by a genetic error that duplicates a section of the X chromosome that includes the MECP2 gene. The MECP2 Duplication Syndrome Fund at RSRT, created in 2010, puts the intellectual and scientific resources of RSRT immediately to work for the MDS community.

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Rett Syndrome Genetic Medicines Summit 2023

In 2023 we hosted the first meeting of its kind with biopharma companies front and center. The Summit gathered basic scientists, pre-clinical, clinical, and regulatory experts to foster discussion, refine expectations, and identify best practices to expedite genetic medicine development.

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$40M