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Press Release

Rett Syndrome Research Trust Launches the Rett Syndrome Global Registry: A Pioneering Parent-Reported Platform Designed to Expedite the Development of Genetic Medicines

Media Contact
Monica Coenraads
Chief Executive Officer
Rett Syndrome Research Trust
November 9, 2023

The Rett Syndrome Research Trust (RSRT) is excited to announce the launch of the Rett Syndrome Global Registry, a fully-remote platform for parents to share their knowledge and experiences caring for loved ones with Rett syndrome. Inclusivity is a vital component of the Rett Global Registry. It is accessible to families regardless of geographic location, access to medical facilities, or socioeconomic status. Designed to advance the development of genetic medicines, the Rett Global Registry adheres to FDA guidance documents and complies with regulatory requirements. Biopharmaceutical companies pursuing therapeutic programs for Rett are urgently waiting to analyze data collected from the Rett Global Registry. Parents of children and adults with Rett syndrome are encouraged to register as soon as possible.

With the increasing role of the patient voice in drug development the Rett Global Registry will capture quality of life and symptom burden at home. The registry has flexible research capabilities for endpoint development and the potential to stand in as an external control arm for clinical trials. More than 300 dedicated parents have contributed foundational datasets, ensuring its immediate utility. “We are incredibly encouraged by the level of interest and support we’ve received from regulatory policy makers, biopharmaceutical companies, and families. We are excited to be working with companies to aid their clinical development so early in the life cycle of the Rett Global Registry,” said Jana von Hehn, PhD, Chief Scientific Officer, Principal Investigator and Director of the Rett Global Registry. Key highlights of the Rett Global Registry include:

  1. Powered by Matrix from Across Healthcare: The Rett Global Registry is hosted on the Matrix platform from Across Healthcare. This state-of-the-art platform offers robust capabilities for securely collecting and managing critical patient information, ensuring data integrity and regulatory compliance while providing unique care management tools to parents.
  2. Multilingual: The Rett Global Registry is available in nine languages, promoting inclusivity and expanding its reach to define care strategies globally.
  3. Regulatory Alignment: RSRT’s dedication to advancing a cure for Rett syndrome is underscored by its choice to adhere to clinical trial database requirements including Title 21 Code of Federal Regulations Part 11, regulatory guidances on patient registries, and meeting with the FDA's Center for Biologics Evaluation and Research (CBER) team during the registry’s development.
  4. Collaboration of Expert Teams: The development of the Rett Global Registry was steered by RSRT’s research team with deep expertise in clinical operations and development. RSRT partnered with expert clinical leads Dr. Bernhard Suter at Baylor College of Medicine, Director of the Blue Bird Circle Rett clinic and investigator in the Neurogene gene therapy trial, and Dr. Cary Fu at Vanderbilt University, Director of the Rett syndrome clinic and lead author on the Consensus guidelines for managing Rett syndrome. In addition, committees for various stakeholders, including parents, clinicians, and biopharmaceutical companies, were instrumental in shaping the registry's structure and objectives.
  5. Part of the CURETT Initiative: RSRT's CURETT initiative (Combining Untapped Resources to Expedite Targeted Therapeutics) is comprised of three complimentary studies: SHARE, CARE and WEAR. The SHARE study captures parent-reported data through the Rett Syndrome Global Registry. The CARE study extracts clinician reported data from medical records. The WEAR study collects objective symptom data from biosensors.
  6. Acknowledgment of Inspirational Contributors: RSRT extends its heartfelt appreciation to several advocacy groups, including FAST, FARA, the Loulou Foundation, Cystic Fibrosis Foundation, Michael J Fox Foundation, and Patients Like Me. Their inspiration and contributions have been instrumental in driving the Rett Global Registry effort forward.

Monica Coenraads, Chief Executive Officer of RSRT, expressed her enthusiasm: "The launch of the Rett Syndrome Global Registry represents a transformative moment in the Rett community. By gathering invaluable parent-reported data from around the world, providing a flexible research arm to facilitate novel endpoint development, and establishing a centralized regulatory compliant infrastructure to support external control arms, we are taking a giant leap forward in expediting the development of curative therapeutics. This registry is a testament to the power parents have to advance a cure.”

To learn more about the registry please visit our website.

About Rett Syndrome Research Trust
RSRT is a nonprofit organization with a highly personal and urgent mission: achieving a cure for Rett syndrome and related disorders caused by defects in the MECP2 gene. Since its founding in 2008, RSRT has awarded more funds than any other Rett organization in the world to leading scientists pursuing targeted research on Rett. RSRT funds and spearheads global scientific and clinical activities advancing the most promising curative approaches. To date, every biopharmaceutical company pursuing a cure for Rett syndrome is doing so because they leveraged discoveries and resources incubated with RSRT funding.