Finding Better Treatments for Rare Disease
About 7,000 rare diseases affect 30 million Americans. Two-thirds of those affected are children.
At Sanford Research, we’re working to improve treatment of rare and pediatric diseases – neurodegenerative diseases including Batten disease, ciliary-associated disorders, kidney diseases and many more.
Our work includes:
- Understanding the genetic, cellular and molecular development of organs and disease
- Developing and testing effective therapies in the lab and clinical trials
By uncovering the causes of rare and childhood disorders, we can create better ways to treat them.
The Pediatrics and Rare Diseases Group supports:
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