NEW YORK–(BUSINESS WIRE)–Kantar Health, a leading global healthcare data, analytics and research provider, and the Cure & Action for Tay-Sachs (CATS) Foundation presented the latest version of the GM2 Disease Registry at the 2020 NORD Rare Diseases & Orphan Products Breakthrough Summit, held October 8-9.
The GM2 Disease Registry (GM2DR) was established in 2015 to increase awareness of and support for clinical research on Tay-Sachs and Sandhoff Disease. In the United States a rare disease is defined as a condition that affects fewer than 200,000 people. Tay-Sachs affects 1 in 320,000 people and Sandhoff affects 1 in 300,000 people.
Managed by CATS, GM2DR leverages the Open App system, which includes patient data such as genotype and key disease milestones including seizure activity and feeding intervention.
There are currently 85 TS and 29 SD patients included in the registry, making it a valuable research platform of real-world data that can provide a deeper understanding of the natural history and the longitudinal progression of these rare diseases.
Real world evidence is anticipated to be used for regulatory and health technology assessment evaluations of emerging therapies.
Daniel Lewi, Co-Founder and CEO of The CATS Foundation, commented: “Our disease registry is built on patient and carer data, providing a powerful tool for those seeking a better understanding of Tay-Sachs and Sandhoff disease. Working together with Kantar Health, we now have an even stronger resource at our fingertips that allows us to gain a deeper understanding of patients and support the clinical development of new compounds which may one day provide a cure for these devastating diseases.”
“Our partnership with the CATS Foundation is a great opportunity to demonstrate the importance of understanding Tay-Sachs and Sandhoff from the patient and carer perspectives, helping life science companies to identify and develop the most meaningful outcomes,” said Michael Fronstin, Global Head of Innovation, Real-World Evidence, Kantar Health.
About KANTAR HEALTH:
Kantar Health, a division of Kantar, provides data, analytics and research to the life sciences industry. As real-world evidence and commercialization partners, Kantar Health enables clients to bring safe and effective treatment to patients around the world. The team has expertise across a myriad of therapy areas, including unique thought leadership in oncology and rare diseases. For more information on how to optimize commercial launch plans, demonstrate real-world effectiveness and safety, and build ongoing value substantiation to unlock your full potential, visit www.kantar.com/health. Kantar helps clients understand people and inspire growth.
About THE CURE & ACTION FOR TAY-SACHS (CATS) FOUNDATION
The Cure & Action for Tay-Sachs (CATS) Foundation is a registered charity based in the UK which supports children and adults affected by Tay-Sachs and Sandhoff disease. We are the founding member of the European Tay-Sachs and Sandhoff Charity Consortium (ETSCC) and have helped drive forward the research and clinical trials for these rare diseases.