A key IAMI collaborative partnership is with the Center for Pediatric Genomic Medicine (CPGM) at Children's Mercy Kansas City (CMH), established in 2011, and the Kansas Intellectual and Developmental Disabilities Research Center (KIDDRC) at the University of Kansas.
Partnership: The CPGM is among the first of its kind with a pediatric focus, providing clinical genomics services and genomic research in rare, inherited diseases in children. The KIDDRC, supported by the National Institute for Child Health and Human Development (NICHD) for nearly 50 years, conducts research aimed at the causes, prevention and treatment of intellectual and related developmental disabilities. Building upon these regional strengths and capabilities in rare disease genomic medicine and intellectual and developmental research, IAMI collaborated with CPGM and KIDDR to develop the Gene-to-Drug-to-Children (GDC) strategy.
Success Story: The GDC strategy allows researchers to characterize the biology of a genomic discovery, identify and validate drug targets, discover new agents, exploit opportunities to repurpose existing drugs and advance promising new treatments to children suffering from rare diseases. With more than 7,500 single gene disorders reported, GDC provides a framework for selecting initial rare disease projects to demonstrate proof of concept based on unmet medical need, patient value and feasibility criteria and characteristics. Frontiers: Heartland Institute for Clinical and Translational Research patient and community engagement lead, Dr. Kim Kimminnau, Kelly Ranallo, co-founder and president of the Turner Syndrome Global Alliance and IAMI director Scott Weir Pharm.D., Ph.D., have brought together Frontiers partners University of Kansas Medical Center, CMH, University of Missouri-Kansas City, the Stowers Institute for Medical Research, the Global Genes Project, the Turner Syndrome Global Alliance and the Kansas City area chapter of the Cystic Fibrosis Foundation to rally the Kansas City region in support of T1 through T4 rare disease translational research.
