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. To learn more, please visit our website.

Media Contact

Monica Coenraads, Rett Syndrome Research Trust, 1 2034450041, [email protected], https://reverserett.org/

SOURCE Rett Syndrome Research Trust