Pediatrics and Rare Diseases
There are over 7,000 different rare diseases that exist today. Rare diseases affect approximately 30 million Americans or one in 10 people. Of the 30 million Americans affected, two-thirds are children and less than one percent of the diseases have an FDA approved treatment. Almost 80 percent of rare diseases have a genetic component that causes the disease and half of these diseases have no devoted support or research.
The Pediatrics and Rare Diseases Group at Sanford Research is dedicated to improving the treatment of rare and pediatric diseases and becoming a global leader in the development of diagnostic and therapeutic approaches. Basic research projects seek to understand the cellular and molecular levels of organ development along with the genetic, molecular and cellular mechanisms of disease. We can use the knowledge of disease mechanisms to develop and test effective therapies in the lab and in clinical research trials. Strengths, expertise and interests of this group include neurodegenerative diseases including Batten disease, ciliary associated disorders, kidney diseases and many more.
This group includes the Children’s Health Research Center, the National Institutes of Health-funded Pediatric Research Center of Biomedical Research Excellence and the Coordination of Rare Diseases at Sanford (CoRDS).