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How Families and the Rett Syndrome Global Registry Will Help Cure Rett

The Rett Syndrome Global Registry is almost ready for launch! To build the Global Registry we had to determine what knowledge gaps exist, define how the Global Registry could best fill them, and work with a top-quality registry design team. While creating the registry we’ve gathered input from Rett families, researchers, biopharmaceutical companies, and disease advocacy groups so that it meets the needs of everyone who has a stake in Rett research. After several years of learning, collaborating, strategizing, and building, the Rett Syndrome Global Registry is about to leap from dream to reality.

We expect the Rett Syndrome Global Registry to revolutionize how we care for individuals with Rett syndrome. Our vision at RSRT is to create a next-generation registry that is driven by Rett caregivers, tailored to make the best use of the information you share about your loved one, integrates clinical data that may be available, and propels research forward by giving our biopharmaceutical partners data to maximize the efficiency of their Rett syndrome drug development programs. If you want early access and to be one of the first Rett families to share information, there is still time to become a Global Registry Pioneer!

For those of you who are familiar with our approach to curing Rett, you know that we don’t shy away from things that haven’t been done before – at RSRT it’s go big or go home. As we prepare to launch, I would like to highlight the innovations and vision of the Global Registry:

The Global Registry will drive research forward unlike anything we’ve done before. In addition to being the most comprehensive collection of caregiver-reported data, the registry will integrate data from sources such as our digital natural history study, data captured from biosensors and the RSRT biorepository, and information from other studies and initiatives. The knowledge generated from multiple measures of Rett will help researchers and biopharmaceutical companies achieve a more complete understanding of the disorder, which will better support the exciting drug development efforts underway. We’ve also met with the FDA to give them a high-level overview, and we will continue to meet with them to receive advice and guidance on their expectations for the registry to appropriately support biopharmaceutical Rett programs.

The Global Registry is for everyone, and what we learn from it belongs to all of us. Any family anywhere in the world can enter data about their loved one and participate in research through the registry. You don’t need to visit a Rett clinic or doctor’s office to join, and the registry can be accessed online, from the comfort of your home.

Because the Global Registry is administered by RSRT, anyone from the community of Rett families and researchers, and any drug development company will be able to access the rich information that it holds. Because the registry database is built from flexible, searchable software, it will be easy to run research inquiries and provide the exact data that labs and companies need. The Global Registry will be a valuable community resource whose quality improves with every family that joins.

The Global Registry empowers caregivers. You know your child with Rett better than anyone, and that knowledge is powerful. When you join the Global Registry, you’ll provide your untapped, valuable information. You’ll be able to contribute to a growing dataset that captures how Rett changes over time, track your loved one’s symptoms, therapies, and skills, as well as log medications and therapies, all of which can be shared with clinicians. As the registry grows so will its value. In time you'll be able to tap into the knowledge of hundreds or thousands of parents. You’ll be able to find answers to your questions by querying the registry and by connecting with other Rett families inside the registry.

The Global Registry will provide a comprehensive view of Rett syndrome. In addition to helping you better track how your loved one is doing, you will also get an overview of the global Rett community through the eyes of parents. Through community summaries you’ll see snapshots of how Rett families are geographically distributed, what are the most prevalent mutations, and how frequently different treatments are used by other families, among many other community facts and figures. As more people join we will all get a more complete picture of Rett. Families that join the Ciitizen digital natural history study by consolidating their medical records will be contributing even more comprehensive data to Rett research.

Together, comprehensive medical records and parent experiences will help us understand how symptom severity reported in the clinic impacts how families feel and function at home. Furthermore, currently there is no good way to know what the true cost of caring for someone with Rett syndrome is. The digital natural history study will help us get a better idea of that, which can help biopharmaceutical companies, the FDA , and others understand the burden of Rett syndrome on the health care system and on families.

The approach of the Rett Syndrome Global Registry is brand new and it is BIG – it’s one of the biggest things we’ve ever done in service of identifying and accelerating a cure. I look forward to sharing so much more with you as we get closer to the launch in early 2022! We hope you will participate and contribute your experience in our mission to cure Rett.

Read the press release about RSRT's vision for the Global Registry.