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Neurogene Announces Rett Syndrome Gene Replacement Program
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RSRT Town Hall:
When will there be a cure for Rett syndrome?
April 13, 2022
1-2:30 p.m. EDT
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Welcome to the Rett cure community

The fierce love we feel for our children drives our singular focus: a cure for Rett syndrome. We are advancing six genetic-based strategies that attack the root cause of Rett from every angle. Our incredible progress is made possible by the support, passion, and commitment of Rett families around the world.

We are the only Rett nonprofit organization solely focused on cure. We are the only organization with a comprehensive plan to achieve a cure and with staff that has decades of industry experience in drug and business development. We have funded more research than any other Rett organization worldwide.

We will not rest until we have a cure.

Join the Rett cure community to be part of the progress.

Recently diagnosed?
We’re here to help

Getting a Rett syndrome diagnosis is one of the hardest things you’ll ever face. We know this first-hand. We’re parents too. Emotions can run the gamut and it can all seem overwhelming. You’re worried about your child and your family. You are reading everything you can get your hands on but you are not sure how to interpret it or if it's relevant.

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CURE 360

Attacking Rett Syndrome
From Every Angle

There are no treatments specific to Rett. The drugs currently in use provide palliative care at best. We believe a genetic disease requires a genetic solution. Our staff and advisors developed our CURE 360 blueprint, which consists of six research strategies that attack Rett from every angle. Multiple shots on goal mitigate risk and maximize the likelihood of success.

Because of our strategic approach there are now seven biopharmaceutical companies with genetic-based treatments for Rett in their drug development pipeline.

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Take Action

Every donation made to RSRT has occurred because a Rett family took action. If your child, grandchild, niece, godchild, or child of your close friend has Rett syndrome, we need your help. Please join our effort.

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Rett Syndrome Global Registry

Imagine Revolutionizing Your Child’s Clinical Care

In 2022 RSRT will launch the Rett Syndrome Global Registry, a virtual, caregiver-driven registry where you can easily enter information about your loved one, track symptoms and therapies to make informed decisions about their care, and participate in research for a cure. The power of the Global Registry will come from the quality and volume of the information that Rett families share. With aggregated, anonymized data from across the global Rett community, biopharmaceutical companies will be able to design more efficient drug studies and get effective drugs to market sooner.

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MECP2 Duplication Syndrome

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. The Fund exclusively supports research aimed at reversing MDS. 100% of every dollar contributed is invested in research — not a single penny goes to overhead.

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